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Research projects
CANADA
Alberta
CALGARY
Identifying Cellular and Molecular Modifiers of Muscular Dystrophy
University of Calgary
Department of Biochemistry and Molecular Biology
CANADA
Ontario
OTTAWA
Differential role of myogenic regulatory factors in establishing muscle-specific gene expression
Ottawa Hospital Research Institute
Sprott Center for Stem Cell Research
CANADA
Ontario
OTTAWA
Satellite Stem Cells from Skeletal Muscle for the Treatment of Neuromuscular Disease
Ottawa Hospital Research Institute
Sprott Center for Stem Cell Research
CANADA
Ontario
OTTAWA, ONTARIO
SIX family transcription factors in adult muscle regeneration.
University of Ottawa
Faculty of Medicine -
FRANCE
ILE-DE-FRANCE
PARIS
Development of an innovative magnetic resonance imaging protocol for the in-depth evaluation of the structure of skeletal muscle tissue in patients with muscular dystrophy
Institut de Myologie - Hôpital Pitié-Salpêtrière
Laboratoire d'imagerie et de spectroscopie par RMN
FRANCE
OCCITANIE
TOULOUSE
Intermuscular adipose tissue as a trigger of muscle wasting?
Institut des Maladies Métaboliques et Cardiovasculaires
Laboratoire de recherche sur les obésités
ISRAEL
ISRAEL
REHOVOT
Mechanical signals transduced downstream of the LINC complex-mediated muscular dystrophies
Arnold R. Meyer Institute of Biological Sciences, Weizmann Institute of Science
Department of Molecular Genetics
ITALY
EMILIA ROMAGNA
ADDRESS: NOT PROVIDED - IT
Study of the multiple functions of Nfix in Muscular Dystrophies: a focus on macrophage biology
Institution: Information not provided - IT
ITALY
LAZIO
ROMA
Characterization of pathways regulating cell cycle and muscle-specific transcription during myogenic differentiation: therapeutical implications for muscle regeneration
Parco Scientifico Biomedico di Roma San Raffaele
Laboratorio di Endocrinologia e Metabolismo Molecolare
ITALY
LOMBARDIA
MILANO
Drug repurposing of ERK inhibitors to target the transcription factor Nfix in dystrophic muscles: development of a new proof-of-concept study to hinder Muscular Dystrophies
Università degli Studi di Milano - Scienze Biomolecolari e Biotecnologiche
Dipartimento di Bioscienze
ITALY
PIEMONTE
TORINO
GLUD1 as a potential target in Muscular Dystrophy
Università degli Studi di Torino
Dipartimento di Biotecnologie Molecolari e Scienze Della Vita
JAPAN
JAPAN
OSAKA
Identification of factors inducing MuSC expansion from overloaded muscle - JP
Osaka University Graduate School of Medicine
SPAIN
Cataluña
BARCELONA
Effect of Nintedanib in muscle fibrosis in a mouse model of sarcoglycanopathy and in the function of human and mouse FAP cells
Hospital de la Santa Creu i Sant Pau
SWITZERLAND
Suisse Alémanique
SCHWERZENBACH
Development of novel stem cell-based models to study myogenesis
Bewegung und Gesundheit Departement Gesundheitswissenschaften und Technologie
Department of Health Sciences and Technology - Laboratory of Regenerative and Movement Biology
ESTONIA
Tartu
TARTU
IDOLS-G: Improved diagnostic output in large sarcomeric genes - EE
Department of Clinical Genetics - University of Tartu
Department of Clinical Genetics
FINLAND
Finland
HELSINKI
IDOLS-G: Improved diagnostic output in large sarcomeric genes -FI
Folkhälsan
Folkhälsan Research Center
FRANCE
GRAND-EST
ILLKIRCH-GRAFFENSTADEN
IDOLS-G: Improved diagnostic output in large sarcomeric genes - FR
Institut de génétique et de biologie moléculaire et cellulaire - IGBMC
Département Médecine translationnelle et neurogénétique
HUNGARY
Közép-Magyarország
BUDAPEST
IDOLS-G: Improved diagnostic output in large sarcomeric genes - HU
Semmelweis University
Department of Biophysics and Radiation Biology
ITALY
TOSCANA
SESTO FIORENTINO (FIRENZE)
IDOLS-G: Improved diagnostic output in large sarcomeric genes - IT
Università degli Studi di Firenze
Dipartimento di Biologia
NETHERLANDS
Noord-Holland
AMSTERDAM
IDOLS-G: Improved diagnostic output in large sarcomeric genes - NL
Amsterdam UMC, locatie VUmc
Afdeling Fysiologie
SPAIN
Cataluña
BARCELONA
IDOLS-G: Mejora del rendimiento diagnóstico en genes sarcoméricos de gran tamaño - ES
Vall d'Hebron Institut de Recerca VHIR
Grupo de investigación en neurología pediátrica
AUSTRALIA
Victoria
MELBOURNE
Pre-clinical drug screen for LAMA2 congenital muscular dystrophy
Monash University
Neuromuscular Disease Research Group
AUSTRIA
WIEN
WIEN
Molecular mechanisms of plectin-related muscular dystrophy
Medizinische Universität Wien
Zentrum für Anatomie und Zellbiologie
CANADA
Ontario
HAMILTON
Role of Xin, an actin-binding protein, in satellite cells and muscular dystrophies
McMaster University
Department of Pathology and Molecular Medicine
CANADA
Québec
JONQUIÈRE
Development of a questionnaire to assess the severity of dysphagia in oculopharyngeal muscular dystrophy
CSSS - Centre de santé et de services sociaux de Jonquière
Centre de réadaptation Le Parcours
CANADA
Québec
QUÉBEC
Multifunctional properties of osteoprotegerin in muscular dystrophy and muscle repair
CHUQ - (CHUL) Centre hospitalier de l'Université Laval
Axe Neuosciences, CHUL
CANADA
Québec
SHERBROOKE
Characterization of a novel function for PABPN1: the product of the oculopharyngeal muscular dystrophy disease gene
Université de Sherbrooke
Département de biochimie
FRANCE
AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR
Characterization of Liver-induced allo-specific CD8 regulatory T lymphocytes
Institution: Information not provided - FR
FRANCE
AUVERGNE-RHONE-ALPES
TORONTO
Safety and efficacy of a possible epigenetic therapy for FSHD muscular dystrophy
Ontario Institute for Cancer Research
Therapeutic Innovation and Drug Discovery
FRANCE
GRAND-EST
ILLKIRCH-GRAFFENSTADEN
Physiopathology of muscle atrophy in myotonic dystrophy
Institut de génétique et de biologie moléculaire et cellulaire - IGBMC
Département Médecine translationnelle et neurogénétique
FRANCE
ILE-DE-FRANCE
FONTENAY-AUX-ROSES
Functional analysis of separase-dependent lamins' regulation in AD-EDMD - FR
Institut de biologie François Jacob
Laboratoire Réparation Et Vieillissement - LREV
FRANCE
ILE-DE-FRANCE
PARIS
Caveolin-assisted sphingolipid transport to the plasma membrane in epidemiology of muscle dystrophies
CLCC Institut Curie
Institut Curie
FRANCE
ILE-DE-FRANCE
PARIS
Schwartz-Jampel syndrome and perlecan deficiency: test of new chemical therapeutic approaches on patients cells and study of physiopathological mechanisms in murine model
Institut du Cerveau et de la Moelle épinière (ICM) - Hôpital Pitié-Salpêtrière
Equipe "Bases moléculaires, physiopathologie et traitement des maladies neurodégénératives"
FRANCE
ILE-DE-FRANCE
PARIS
Innovative bio-inspired cell and tissue models of genetic dilated cardiomyopathy for disease modelling and drug discovery
Université Pierre et Marie Curie - Paris 6 - UPMC
Adaptation Biologique et Vieillissement
FRANCE
ILE-DE-FRANCE
VERSAILLES
The impact of residual dystrophin on the natural history of dystrophinopathies.
Université de Versailles Saint-Quentin
UMR U1179 - Handicap neuromusculaire : Physiopathologie, Biothérapie et Pharmacologie appliquées
FRANCE
OCCITANIE
MONTPELLIER
Unravelling molecular mechanisms of DMD gene splicing regulation and their roles as disease modifiers in Duchenne muscular Dystrophy.
Institut Universitaire de Recherche Clinique EA 7402 Université de Montpellier
Laboratoire de Génétique de Maladies Rares
FRANCE
OCCITANIE
MONTPELLIER
New therapeutic strategies development for oculopahryngeal muscular dystrophy using a drosophila model
Institut de Génétique Humaine (IGH)
Département Génétique et Développement
FRANCE
OCCITANIE
MONTPELLIER
riboOPMD: Ribosomal and small non-coding RNAs in oculopharyngeal muscular dystrophy - FR
Institut de Génétique Humaine (IGH)
Département Génétique et Développement
FRANCE
OCCITANIE
MONTPELLIER
Role of oxidative stress and endoplasmic reticulum stress in OPMD: molecular mechanisms and pharmacological treatments.
Institut de Génétique Humaine (IGH)
Département Génétique et Développement
FRANCE
PROVENCE-ALPES-COTE D'AZUR
MARSEILLE
Characterization of the phenotypic variability in FSHD families for assisting clinical research
CHU de Marseille - Hôpital de la Timone
Service de Neurologie - Maladies neuromusculaires et SLA
FRANCE
PROVENCE-ALPES-COTE D'AZUR
NICE
French National Registry for FacioScapuloHumeral muscular Dystrophy (FSHD)
CHU de Nice - Hôpital Pasteur
Service Neurologie - Système Nerveux Périphérique et Muscles / Sclérose Latérale Amyotrophique
FRANCE
PROVENCE-ALPES-COTE D'AZUR
NICE
Resolve FSHD: clinical trial readiness to solve barriers to drug development in FSHD
CHU de Nice - Hôpital Pasteur
Service Neurologie - Système Nerveux Périphérique et Muscles / Sclérose Latérale Amyotrophique
FRANCE
PROVENCE-ALPES-COTE D'AZUR
NICE
New clinical outcome measures to evaluate non-ambulant FSHD patients
Faculté de médecine de Nice Sophia-Antipolis
CHU Nice
GERMANY
Bayern
MARTINSRIED/PLANEGG
EpiThe4FSHD: Safety and efficacy of a possible epigenetic therapy for FSHD muscular dystrophy -DE
BioMedical Center - BMC
Department Molecular Biology
ISRAEL
ISRAEL
ADDRESS: NOT PROVIDED - IL
Epigenetic analysis of myonuclei defective in nuclear envelope components in Drosophila muscles as a model for studying Emery-Dreifuss muscular dystrophy
Institution: Information not provided - IL
ITALY
EMILIA ROMAGNA
ADDRESS: NOT PROVIDED - IT
Bone phenotype in Duchenne muscular dystrophy: unveiling the role of LCN2 and implications for therapy.
Institution: Information not provided - IT
ITALY
LAZIO
ROMA
Role of miR-200c in dystrophic muscle regeneration of mdx mice and DMD patients
Istituto Dermopatico dell'Immacolata - IRCCS
I Divisione Dermatologia
ITALY
LAZIO
ROMA
In vivo characterization of miR-200c in regeneration of dystrophic skeletal muscles in mdx mice
Sapienza Università di Roma
Dipartimento di Genetica e Biologia Molecolare
ITALY
LAZIO
ROMA
The RAMYD (risk of arrhythmias in Myotonc Dystrophy Type I) study: phase II
Università Cattolica del Sacro Cuore
Istituto di Anatomia Umana e Biologia Cellulare
ITALY
LAZIO
ROMA
Characterization of the Fibroadipogenic progenitors in Facioscapulohumeral dystrophy (FSHD) patients and their role in the pathogenesis of the disease
Università Cattolica del Sacro Cuore
ITALY
LOMBARDIA
MILANO
Dissecting the role of chromatin structure and alternative splicing in the development and pathology of Emery-Dreifuss Muscular Distrophy
IFOM - Istituto FIRC di Oncologia Molecolare
Firc Institute of Molecular Oncology (IFOM)
ITALY
LOMBARDIA
MILANO
An animal model to develop therapeutic strategies for Facioscapulohumeral Muscular Dystrophy (terminated)
IRCCS Ospedale San Raffaele
Fondazione Centro San Raffaele del Monte Tabor - Divisione di Medicina Rigenerativa Cellule Staminali e Terapia Genica (DMR)
ITALY
LOMBARDIA
MILANO
Characterization of a novel inhibitor of DUX4 expression
IRCCS Ospedale San Raffaele
Fondazione Centro San Raffaele del Monte Tabor - Divisione di Medicina Rigenerativa Cellule Staminali e Terapia Genica (DMR)
ITALY
LOMBARDIA
MILANO
Modulation of the cyclin inhibitor p27 to ameliorate Merosin Deficient Congenital Muscular Dystrophy (MDC1A)
IRCCS Ospedale San Raffaele
Istituto di Neurologia Sperimentale - Unità di Ricerca Rigenerazione Neuromuscolare
ITALY
LOMBARDIA
MILANO
Dissecting the epigenetic features at the basis of Facioscapolohumeral Distrophy (terminated)
Università degli Studi di Milano - Biologia e Genetica per le Scienze Mediche
Dipartimento di Biologia e Genetica per le Scienze Mediche
ITALY
SICILIA
TROINA
Mutational analysis of the genes POMGnT1, FKRP, and LARGE FKTN in congenital muscular dystrophy type 1A
IRCCS OASI Maria Santissima
Laboratorio di Diagnosi Genetica
ITALY
TOSCANA
PISA
EpiThe4FSHD: Safety and efficacy of a possible epigenetic therapy for FSHD muscular dystrophy - IT
Azienda Ospedaliero Universitaria Pisana - Ospedale S. Chiara
UOC Neurologia
ITALY
VENETO
PADOVA
La spermidina: un nuovo candidato per il trattamento delle miopatie da carenza di collagene VI
Università degli Studi di Padova- Polo A.Vallisneri
Dipartimento di Istologia, Microbiologia e Tecnologie Mediche
NETHERLANDS
Utrecht
ADDRESS: NOT PROVIDED - NL
Evaluation of dysphagia in inclusion body myositis and oculopharyngeal muscle dystrophy by combining novel ultrasound and real-time MRI
Institution: Information not provided - NL
NETHERLANDS
Zuid-Holland
LEIDEN
RNA processing role in muscle degeneration opens therapeutical options for adult myopathies
LUMC - Leids Universitair Medisch Centrum
Afdeling Humane Genetica
PORTUGAL
SUL
LISBOA
Understanding the responses of fetal muscle stem cells and myoblasts to their niche in a mouse model for LAMA2-CMD
Faculdade de Ciências da Universidade de Lisboa
SPAIN
Cataluña
BARCELONA
Advanced gene editing technologies to restore LAMA2 on merosin-deficient congenital muscular dystrophy type 1A
Universitat Pompeu Fabra. Campus del Mar
Grupo de Biología Sintética Traslacional
SPAIN
Cataluña
BARCELONA
Development of a novel gene therapy strategy and clinical trial readiness of a cohort of patients with merosin-deficient congenital muscular dystrophy
Vall d'Hebron Institut de Recerca VHIR
Grupo de investigación en terapia celular y genética
SPAIN
Cataluña
ESPLUGUES DE LLOBREGAT
Personalized medicine for congenital muscular dystrophies: development of advanced therapies, physiological models and precise diagnostic tools
Fundació Sant Joan de Déu - Institut de Recerca Sant Joan de Déu
Grupo de Investigación aplicada en enfermedades neuromusculares
SPAIN
Madrid
MADRID
Study of the potential of CRISPR/Cas9 technology for the treatment LMNA-associated congenital muscular distrophy
ISCIII - Instituto de Salud Carlos III
Instituto de Salud Carlos III
SPAIN
País Vasco
SAN SEBASTIÁN
Evaluation of CRISPR-dCas13 system as a precision medicine approach for neurodegenerative disorders: in vitro studies and in an animal model of Spinal Muscular Atrophy (SMA)
Instituto de Investigación Sanitaria Biodonostia
Área de Neurociencias
SWEDEN
Region Skåne
LUND
Laminins and Cib2 in congenital muscular dystrophy
Lund University
Department of Experimental Medical Science
SWEDEN
Region Stockholm
STOCKHOLM
The functional organisation of the brain
Karolinska Institutet - Solna
Department of Clinical Neuroscience (CNS)
SWEDEN
Region Stockholm
STOCKHOLM
Genetic and molecular mechanisms in familial visual impairment
Karolinska Institutet - Solna
Department of Molecular Medicine and Surgery (MMK)
UNITED KINGDOM
Greater London
LONDON
DUX4 in FSHD: pathophysiology and therapeutic approaches
University College London - UCL Darwin Building
UCL GOS Institute of Child Health
UNITED KINGDOM
Merseyside
LIVERPOOL
INPP5K-mediated congenital muscular dystrophy models of motor neuron branching and function.
University of Liverpool
UNITED KINGDOM
Surrey
EGHAM
Establishing a mouse model of disease to test combined antisense oligonucleotides and AAV systemic gene therapy as new treatment for Oculopharyngeal muscular dystrophy
Royal Holloway, University of London
Centre of Gene and Cell Therapy
UNITED KINGDOM
Surrey
EGHAM
Identifying metabolic signatures of pathology in muscle and blood to identify biomarkers of oculopharyngeal muscular dystrophy
Royal Holloway, University of London
School of Biological Sciences
UNITED STATES
Pennsylvania
PITTSBURGH
Therapeutic genetics and disease modeling in LAMA2-CMD
University of Pittsburgh
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Magnetic resonance imaging and spectroscopy biomarkers for facioscapulohumeral muscular dystrophy
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Interventions in genetic counseling
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Genetic and epigenetic mechamisms of fshd pathogenesis
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Molecular and therapeutic mechanisms of a new model of congenital muscular dystrophy
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Muscle-specific regulation of pabpn1 expression
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Myonuclear homeostasis in craniofacial muscles
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Project 1: preclinical development of surrogate gene therapy using galgt2
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Project 2: dux4 inhibition with non-coding rnas as a therapeutic strategy for facioscapulohumeral muscular dystrophy (fshd)
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Regulation of subcellular organization in skeletal muscle
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Safety and feasibility study of transvenous limb perfusion with normal saline in
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Translational and pre-clinical studies of aav-mediated gene therapy of muscular
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Health endpoints and longitudinal progression in congenital myotonic dystrophy (help-cdm)
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Therapeutic strategies for microsatellite expansion diseases using rna-targeting crispr/cas
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Mechanisms of cognitive deficits in dystroglycanopathies
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Genetic modifiers of fshd
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Animal models of fshd for therapy
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Biomarker discovery in muscles from fshd patients
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Fshd disease biomarkers
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Myogenesis studies for fshd biomarkers
Institution: Information not provided - US
AUSTRIA
WIEN
WIEN
Long-Term Observational Study of Translarna Safety and Effectiveness in Usual Care - AT
Gottfried von Preyer'sches Kinderspital
Abteilung für Kinder- und Jugendheilkunde
BELGIUM
ANTWERPEN
ANTWERPEN
A preclinical study to treat neuromuscular diseases caused by mutations in the small heat shock protein HSPB8
University of Antwerp - UA, Campus Drie Eiken
Peripheral Neuropathy Research Group
BELGIUM
ARRONDISSEMENT BRUSSELS-CAPITAL
BRUSSELS
CURE-DM1: In vivo CRISPR/Cas9-mediated correction of triplet nucleotide repeat expansion in DM1
VUB_ Vrije Universiteit Brussel, Campus Jette
Department of Gene Therapy and Regenerative Medicine
CANADA
Alberta
EDMONTON
Targeted delivery of antisense-mediated exon skipping therapy in transgenic pig model of DMD
University of Alberta
Department of Medical Genetics
CANADA
Alberta
EDMONTON
Targeting RNA Splicing for the Treatment of Duchenne Muscular Dystrophy
University of Alberta
Department of Medical Genetics
CANADA
Ontario
MISSISSAUGA
Synaptic physiology and structure of human muscle in a 3D cell culture system: Application to Duchenne's Muscular Dystrophy
University of Toronto Mississauga
Department of Biology
CANADA
Ontario
OTTAWA
Defining the Role and Therapeutic Potential of Pannexin 1 Channels in Duchenne Muscular Dystrophy using Mouse Models and Patients Cells
Children's Hospital of Eastern Ontario
Children's Hospital of Eastern Ontario Research Institute
CANADA
Ontario
OTTAWA
Genetic Regulation of Myogenesis
Ottawa Hospital Research Institute
Sprott Center for Stem Cell Research
CANADA
Ontario
OTTAWA
Stimulating cilia-mediated Hedgehog signaling to restore dystrophin-deficient satellite cell function
Ottawa Hospital Research Institute
Sprott Center for Stem Cell Research
CANADA
Ontario
OTTAWA, ONTARIO
Post-transcriptional Regulation of Utrophin in Skeletal Muscle: Implications for New Therapeutic Strategies for Duchenne Muscular Dystrophy
University of Ottawa
Faculty of Medicine -
CANADA
Ontario
OTTAWA, ONTARIO
Inhibition of HDAC6 as Therapeutic Strategy in Neuromuscular Diseases
University of Ottawa
Faculty of Medicine -
CANADA
Ontario
OTTAWA, ONTARIO
Novel AMPK activators as relevant therapeutics for the treatment of Myotonic Dystrophy type 1 (DM1)
University of Ottawa
Department of Cellular & Molecular Medicine
CANADA
Ontario
OTTAWA, ONTARIO
Role of the RNA-binding protein HuR in Myotonic Dystrophy type 1
University of Ottawa
Department of Cellular & Molecular Medicine
CANADA
Ontario
TORONTO
Growth arrest and osteoporosis in Duchenne muscular dystrophy patients treated with glucocorticoids
Mount Sinai Hospital
Lunenfeld-Tanenbaum Research Institute
CANADA
Québec
ADDRESS: NOT PROVIDED - CA
Role of Calcium Handling Proteins and Calcineurin Signaling in Myotonic Dystrophy type 1
Institution: Information not provided - CA
CANADA
Québec
ADDRESS: NOT PROVIDED - CA
Elucidation of Wnt7a mechanism of action for muscle regeneration
Institution: Information not provided - CA
CANADA
Québec
ADDRESS: NOT PROVIDED - CA
Cardiac involvement in myotonic dystrophy type 1 disease: development of improved diagnoses and therapeutics
Institution: Information not provided - CA
CANADA
Québec
ADDRESS: NOT PROVIDED - CA
Targeting of Histone Deacetylase 6 (HDAC6) in Neuromuscular Diseases
Institution: Information not provided - CA
CANADA
Québec
ADDRESS: NOT PROVIDED - CA
Repurposing drugs that target eEF1A2 to increase translation of utrophin in dystrophic muscle
Institution: Information not provided - CA
CANADA
Québec
JONQUIÈRE
Motor, multisystemic and social participation assessment in myotonic dystrophy type 1 : a 9-year longitudinal study
CSSS - Centre de santé et de services sociaux de Jonquière
Centre de réadaptation Le Parcours
CANADA
Québec
MONTRÉAL
Using specialized pro-resolving mediators to treat Duchenne muscular dystrophy
Centre hospitalier universitaire Sainte-Justine
Centre de recherche - Centre hospitalier universitaire Saint-Justine
CANADA
Québec
MONTRÉAL
Contribution of autophagy in muscle stem cell dysfunction in Duchenne muscular dystrophy
McGill University - McIntyre Medical Sciences Building
Stem Cell Biology and Regenerative Medicine
CANADA
Québec
MONTRÉAL
Strategies for therapy of respiratory muscle failure in muscular dystrophy
Meakins-Christie Laboratories
Meakins-Christie Labs
CANADA
Québec
QUÉBEC
Correction of the dystrophin gene with TAL effector nuclease and the CRISPR system
CHUQ - (CHUL) Centre hospitalier de l'Université Laval
Unité de Génétique humaine, Axe Neurosciences
CANADA
Québec
QUÉBEC
Can laminin-111 be used to treat Duchenne Muscular Dystrophy alone or in combination with myoblast transplantation?
CHUQ - (CHUL) Centre hospitalier de l'Université Laval
Unité de Génétique humaine, Axe Neurosciences
COSTA RICA
COSTA RICA
SAN PEDRO DE MONTES DE OCA
Profil d'expression comparatif de plusieurs tissus dans la dystrophie myotonique
Universidad de Costa Rica
INSTITUTO DE INVESTIGACIONES EN SALUD
CYPRUS
Cyprus
ADDRESS: NOT PROVIDED - CY
LNA/2'OMe mixmers against toxic CUG expanded RNA
Institution: Information not provided - CY
CYPRUS
Cyprus
NICOSIA
Développement de biomarqueurs à base de miARN pour suivre les progrès de la dystrophie myotonique de type I
The Cyprus Institute of Neurology and Genetics
Molecular Genetics, Function & Therapy
FRANCE
AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR
Gene transfer tolerance in combined liver and muscle rAAV gene therapy
Institution: Information not provided - FR
FRANCE
AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR
Validation of non-invasive non-volitional methods for monitoring respiratory muscle function in Duchenne muscular dystrophy
Institution: Information not provided - FR
FRANCE
AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR
Mise en oeuvre clinique des tests prénataux non invasifs pour les dystrophies musculaires de Duchenne
Institution: Information not provided - FR
FRANCE
AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR
Impact of pre-phonation inspiratory volume on speech quality of neuromuscular patients requiring non invasive ventilation
Institution: Information not provided - FR
FRANCE
AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR
Liver Gene Transfer-Induced allogeneic Tolerance for organ transplantation and cell therapy
Institution: Information not provided - FR
FRANCE
AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR
Impact du type d'interface chez les patients neuromusculaires traités par ventilation non invasive nocturne: un essai croisé randomisé
Institution: Information not provided - FR
FRANCE
AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR
Impact of blowpipe training on cough efficiency in neuromuscular patients : a prospective evaluation.
Institution: Information not provided - FR
FRANCE
AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR
Characterization of CASK protein function in the regulation of ion channels in cardiomyocytes and its role in cardiac electrophysiology in vivo
Institution: Information not provided - FR
FRANCE
AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR
Characterization of a new model of mdx mice deficient in the enzyme CD38: towards the protective role on the Ca2+ homeostasis deregulation
Institution: Information not provided - FR
FRANCE
AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR
Small non-coding RNAs of intron origin in Myotonic Dystrophy type 1: innovative biomarkers and candidate drivers of splicing defects
Institution: Information not provided - FR
FRANCE
AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR
Deciphering new non-coding RNAs from introns : An atlas of new biomarkers/targets for DM1 patients
Institution: Information not provided - FR
FRANCE
AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR
Involvement of endo/sarcoplasmic reticulum - mitochondria interaction in the Duchenne muscular dystrophy
Institution: Information not provided - FR
FRANCE
AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR
Dystrophin and neuronal plasticity: towards a possible treatment of the cognitive defects in DMD using novel antisense oligonucleotides
Institution: Information not provided - FR
FRANCE
AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR
Oligodendrocyte dysfunction and myelin abnormalities in a transgenic mouse model of myotonic dystrophy type 1
Institution: Information not provided - FR
FRANCE
AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR
ROLE OF ENDOPLASMIC RETICULUM-PLASMA MEMBRANE CONTACT SITES (ER-PM CS) AND AUTOPHAGY IN DYSFERLINOPATHY AND CAVEOLINOPATHY
Institution: Information not provided - FR
FRANCE
AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR
L'annonce diagnostique d'une maladie neuromusculaire à l'âge adulte. Retentissements psychologiques des processus communicationnels entre médecin et patient
Institution: Information not provided - FR
FRANCE
AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR
Theory of mind training in virtual reality in child-onset Steinert myotonic dystrophy
Institution: Information not provided - FR
FRANCE
AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR
Duchenne Muscular Dystrophy non-viral gene therapy - FR
Institution: Information not provided - FR
FRANCE
AUVERGNE-RHONE-ALPES
BRON
Collaborative program for a systematic, mecanistic and pharmacologic approach of rare neuromuscular diseases
CHU de Lyon HCL - GH Est
Centre de biotechnologie cellulaire
FRANCE
AUVERGNE-RHONE-ALPES
GRENOBLE
Early identification of respiratory exacerbations using NIV device monitoring in slowly progressive neuromuscular disorders
CHU Grenoble Alpes - Site Nord - Hôpital Couple Enfant
Laboratoire EFCR/Pneumologie
FRANCE
AUVERGNE-RHONE-ALPES
LYON
Interplay between cell metabolism and alternative splicing
ENS - Ecole normale supérieure de Lyon
Laboratoire de biologie et modélisation de la cellule - UMR5239
FRANCE
AUVERGNE-RHONE-ALPES
LYON
Amino acid metabolism and alternative splicing
ENS - Ecole normale supérieure de Lyon
Laboratoire de biologie et modélisation de la cellule - UMR5239
FRANCE
AUVERGNE-RHONE-ALPES
LYON
Development and validation study of a Motor Function Measure digitalized playful completion modules
Hospices Civils de Lyon
FRANCE
AUVERGNE-RHONE-ALPES
PRAGUE
Preclinical assessment of clinic ready agents for the treatment of muscular dystrophy and spinal muscular atrophy
Faculty of Medicine and University Hospital Motol
Department of Pediatrics
FRANCE
ILE-DE-FRANCE
CORBEIL-ESSONNES
GMP production for rare diseases
YposKesi (AFM-Telethon-Genethon)
FRANCE
ILE-DE-FRANCE
CRÉTEIL
TRANSLAMUSCLE: An integrated translational program from basic research to biotherapies in stem cells and molecular medicine of the neuromuscular system
Faculté de Médecine de Créteil
Equipe "Biologie du système neuromusculaire"
FRANCE
ILE-DE-FRANCE
CRÉTEIL
An integrated translational program for neuromuscular disorders
Hôpitaux Universitaires Henri Mondor
Centre expert de maladie neuro-musculaire
FRANCE
ILE-DE-FRANCE
EVRY
Pathological modelling of Steinert Myotrophy using human embryonic stem cells carrying the causal mutation
GENOPOLE - Campus 1
I-Stem - Institut des cellules Souches pour le Traitement et l'Etude des maladies Monogéniques
FRANCE
ILE-DE-FRANCE
GARCHES
Patient-ventilator asynchrony in neuromuscular disease: real life evaluation using devices softwares
CHU Paris IdF Ouest - Hôpital Raymond Poincaré
Service de Physiologie-Explorations Fonctionnelles - Consultations Troubles du sommeil
FRANCE
ILE-DE-FRANCE
GIF-SUR-YVETTE
Rescue_ribosome: Rescue Pathways for unrecycled ribosomes
Institut de Biologie Intégrative de la Cellule (I2BC)
Département Biologie des Génomes
FRANCE
ILE-DE-FRANCE
GIF-SUR-YVETTE
Restoration of Ca2+-signalling in mdx mice by targeting the endo-lysosmal two-pore channel (TPC)
Institut des Neuroscieces Paris-Saclay
Code neuronal & perception auditive
FRANCE
ILE-DE-FRANCE
PARIS
Implementation of non-invasive prenatal diagnosis of rare and severe monogenic diseases
AP-HP.Centre - Université de Paris - Hôpital Cochin
Service de Médecine Génomique des Maladies de Système et d'Organe
FRANCE
ILE-DE-FRANCE
PARIS
FRANCE
ILE-DE-FRANCE
PARIS
MYOCITY - A multidimensional single-cell approach to understand muscle dystrophy - FR
CLCC Institut Curie
Labex Dendritic Cell Biology (DCBIOL)
FRANCE
ILE-DE-FRANCE
PARIS
Induced pluripotent stem cells-based strategies and disease modeling to unravel signaling-induced epigenetic networks in healthy and in Duchenne muscle dystrophic muscles: the TGF beta/Wnt regulated histone lysine methylome
CNRS - Centre national de la recherche scientifique
Centre National de la Recherche Scientifique
FRANCE
ILE-DE-FRANCE
PARIS
Decoding central defects in Dystrophinopathies: From diagnostic to remediation
CNRS - Centre national de la recherche scientifique
Centre National de la Recherche Scientifique
FRANCE
ILE-DE-FRANCE
PARIS
Gene therapy in DM1 cells by induction of a TALE Nuclease
Hôpital Necker-Enfants Malades
Service de Génétique Moléculaire
FRANCE
ILE-DE-FRANCE
PARIS
How to contract CAG.CTG repeats in myotonic dystrophy type 1
IMAGINE - Institut des Maladies Génétiques
Laboratoire "Triplets CTG instables et dystrophie myotonique"
FRANCE
ILE-DE-FRANCE
PARIS
Novel satellite cell heterogeneity in healthy and pathological regeneration - FR
Institut Cochin (INSERM U 1016 - CNRS UMR 8104)
Équipe AMPK et kinases apparentées à l'AMPK dans la physiopathologie du diabète et de l'obésité
FRANCE
ILE-DE-FRANCE
PARIS
Nuclear genetic suppressors in yeast models of mtDNA mutations associated to neuromuscular diseases
Institut National de la Santé et de la Recherche Médicale
FRANCE
ILE-DE-FRANCE
PARIS
L'impact de la sénescence cellulaire et de la reprogrammation in vivo dans la dystrophie musculaire de Duchenne
Institut Pasteur
FRANCE
ILE-DE-FRANCE
PARIS
Tricyclo-DNA antisense oligonucleotide treatment for Myotonic Dystrophy
Institut de Myologie - Hôpital Pitié-Salpêtrière
Équipe "Dystrophie Myotonique, Physiopathologie & Biothérapie"
FRANCE
ILE-DE-FRANCE
PARIS
ReCognitION: Recognition and Validation of Druggable Targets from the Response to Cognitive Behaviour Therapy in Myotonic Dystrophy type 1 patients from Integrated -Omics Networks - FR
Institut de Myologie - Hôpital Pitié-Salpêtrière
Équipe "Dystrophie Myotonique, Physiopathologie & Biothérapie"
FRANCE
ILE-DE-FRANCE
PARIS
Collaborative program for a systematic, mecanistic and pharmacologic approach of rare neuromuscular diseases
Institut de Myologie - Hôpital Pitié-Salpêtrière
Centre de Recherche en Myologie
FRANCE
ILE-DE-FRANCE
PARIS
Spatio-Temporal Profile of Cellular Subfractions in Duchenne Muscular Dystrophy
Institut de Myologie - Hôpital Pitié-Salpêtrière
Centre de Recherche en Myologie
FRANCE
ILE-DE-FRANCE
PARIS
sPAM: Developing peptide-based drugs to target ataxin-2 in neuromuscular disease - FR
Institut du Cerveau et de la Moelle épinière (ICM) - Hôpital Pitié-Salpêtrière
Equipe "Traitement de la sclérose latérale amyotrophique : de la génétique au poisson zèbre"
FRANCE
ILE-DE-FRANCE
PARIS
Newly identified non-coding RNAs from alternatively spliced introns in normal and pathological muscle differentiation
Université Paris Diderot
Unité Epigénétique et Destin Cellulaire CNRS UMR7216
FRANCE
ILE-DE-FRANCE
VERSAILLES
Induction of immunological tolerance by dual muscle and liver gene transfer for Duchenne muscular dystrophy
Université de Versailles Saint-Quentin
UMR U1179 - Handicap neuromusculaire : Physiopathologie, Biothérapie et Pharmacologie appliquées
FRANCE
ILE-DE-FRANCE
VERSAILLES
Elucidate the cellular and molecular mechanisms involved in the generation of revertant dystrophin-positive fibers using the dystrophic DmdEGFP-mdx reporter mouse.
Université de Versailles Saint-Quentin
UMR U1179 - Handicap neuromusculaire : Physiopathologie, Biothérapie et Pharmacologie appliquées
FRANCE
ILE-DE-FRANCE
VERSAILLES
Elucidate the molecular mechanism leading to the generation of revertant dystrophin-positive fibers in muscle dystrophic DmdEGFP-mdx reporter mouse
Université de Versailles Saint-Quentin
Laboratoire Handicap neuromusculaire : Physiopathologue, Biotechnologies et Pharmacologies appliquées (END-ICAP) - UMR U1179
FRANCE
NOUVELLE AQUITAINE
POITIERS
Functional characterization of muscle cells derived from healthy and DMD human induced Pluripotent Stem Cells
Université de Poitiers
Laboratoire Signalisation et Transports Ioniques Membranaires
FRANCE
OCCITANIE
MONTPELLIER
Towards a better understanding of the interplay between cis-acting elements and trans factors driving DMD pre-MRNA splicing
IURC - Institut Universitaire de Recherche Clinique
Laboratoire de génétique moléculaire
FRANCE
OCCITANIE
MONTPELLIER
Cardiac dysfunction in Duchenne Muscle Dystrophy children : pathophysiological role of type 2 ryanodine receptor through hiPSC-derived cardimyocytes
Université Montpellier II
Caractérisation de cardiomyocytes DMD et CPVT « patient-specific » dérivés de cellules souches pluripotentes induites
FRANCE
PAYS DE LA LOIRE
NANTES
ExoMS: a 3D printed exoskeleton with springs to enhance upper limb mobility of children with neuromuscular disease
CHU de Nantes
CHU Nantes
FRANCE
PAYS DE LA LOIRE
NANTES
New modulators of the skeletal and cardiac ryanodine receptors
CHU de Nantes
CHU Nantes
FRANCE
PROVENCE-ALPES-COTE D'AZUR
MARSEILLE
Exploring ventricular conduction system structure and function in the regenerating heart and DMD mouse models
IBDM - Institut de Biologie du Développement de Marseille
Équipe Contrôle génétique du développement cardiaque
FRANCE
PROVENCE-ALPES-COTE D'AZUR
MARSEILLE
Modelisation and Therapeutic Approaches for Rare Diseases
Université de médecine Aix-Marseille Université
Centre de Génétique de Marseille (Marseille Medical Genetics - MMG)
FRANCE
PROVENCE-ALPES-COTE D'AZUR
MARSEILLE
A quantitative MRI platform for simultaneous and automatic quantification of fat infiltration and T2 relaxation times in neuromuscular disorders - FR
Université de médecine Aix-Marseille Université
Centre de Résonance Magnétique Biologique et Médicale
FRANCE
PROVENCE-ALPES-COTE D'AZUR
VALBONNE
Role of glia in neurological comorbidities of Duchenne muscular dystrophy - FR
IPMC - Institut de Pharmacologie Moléculaire et Cellulaire
Equipe de Physiopathologie moléculaire des phospholipases A2 & de leurs médiateurs
GERMANY
Bayern
ERLANGEN
Progression study on the characterization of skeletal muscles by magnetic resonance imaging in Duchenne muscular dystrophy
ZSEER - Zentrum für Seltene Erkrankungen Erlangen
GERMANY
Berlin
BERLIN
TAMDMD: Tamoxifen in Duchenne muscular dystrophy - a randomised placebo controlled phase 2 trial - DE
DRK Kliniken Berlin Westend
Klinik für Kinder- und Jugendmedizin
GERMANY
Berlin
BERLIN
Deciphering the role of Prdm16-mediated H3K9 methylation in the control of Fibro-Adipogenic Progenitors identity and skeletal muscle repair -DE
FU Berlin
Institut für Biochemie
ISRAEL
ISRAEL
TEL AVIV
A quantitative MRI platform for simultaneous and automatic quantification of fat infiltration and T2 relaxation times in neuromuscular disorders - IL
University of Tel-Aviv
Dep. of Bio-Medical Engineering
ITALY
EMILIA ROMAGNA
ADDRESS: NOT PROVIDED - IT
PGC1 alpha gene expression regulation and Mitochondrial Biogenesis impairment in Muscular Dystrophies: new molecular signatures for novel therapeutic strategy
Institution: Information not provided - IT
ITALY
EMILIA ROMAGNA
ADDRESS: NOT PROVIDED - IT
Pin1: a new potential target to induce slow fiber conversion in Duchenne Muscular Dystrophy
Institution: Information not provided - IT
ITALY
EMILIA ROMAGNA
ADDRESS: NOT PROVIDED - IT
Immune system involvement in DMD pathology: the role of the immunoproteasome and its therapeutic potential.
Institution: Information not provided - IT
ITALY
EMILIA ROMAGNA
ADDRESS: NOT PROVIDED - IT
Therapeutic relevance of the sphingolipid-metabolizing enzyme acid sphingomyelinase as a new pathogenetic factor in Duchenne muscular dystrophy
Institution: Information not provided - IT
ITALY
LAZIO
ROMA
Towards Precision Medicine with Human Induced Pluripotent Stem Cells for Dystrophin Associated Cardiomyopathy.
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
ITALY
LAZIO
ROMA
Exosome-mediated HDACi/miR-143/STAT3 network in the regulation of satellite cells expansion and muscle regeneration
IRCCS Fondazione Santa Lucia
Laboratorio di Epigenetica e Farmacologia Rigenerativa
ITALY
LAZIO
ROMA
New therapeutic strategies based on FAPs-derived Exosomes in the treatment of Duchenne Muscular Dystrophy
IRCCS Fondazione Santa Lucia
Laboratorio di Epigenetica e Farmacologia Rigenerativa
ITALY
LAZIO
ROMA
Functionalized nanoparticles for targeted genome editing in Duchenne Muscular Dystrophy - IT
IRCCS Fondazione Santa Lucia
Laboratorio di Epigenetica e Trasduzione del Segnale
ITALY
LAZIO
ROMA
Role of Dystrophin-associated protein complex (DPC) in intracellular signaling and trafficking pathways
ISS - Istituto Superiore di Sanità
Centro Nazionale Malattie Rare
ITALY
LAZIO
ROMA
RNA-based studies of Duchenne Muscular Dystrophy: post-transcriptional control and role of non coding RNAs in normal and dystrophic muscle development
Sapienza Università di Roma
Dipartimento di Biologia e Biotecnologie "Charles Darwin"
ITALY
LAZIO
ROMA
Role of glia in neurological comorbidities of Duchenne muscular dystrophy - IT
Sapienza Università di Roma
FISIOLOGIA E FARMACOLOGIA "VITTORIO ERSPAMER"
ITALY
LAZIO
ROMA
Cellular network driving neuromuscular junction stability
Sapienza Università di Roma
Università degli Studi di Roma "La Sapienza"
ITALY
LAZIO
ROMA
Analysis of the DM2 pathogenic mechanisms using Drosophila as model system
Sapienza Università di Roma - Dip. di Biologia e Biotecnologie Charles Darwin
Dipartimento di Biologia e Biotecnologie - Charles Darwin
ITALY
LOMBARDIA
BRESCIA
Therapeutic potential of Amniotic Mesenchymal Stromal Cells and their released extracellular vesicles in the treatment of Duchenne Muscular Dystrophy
HFP Fondazione Poliambulanza Istituto Ospedaliero
ITALY
LOMBARDIA
MILANO
Novel satellite cell heterogeneity in healthy and pathological regeneration - IT
Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico
Laboratorio Cellule Staminali
ITALY
LOMBARDIA
MILANO
HMGB1 Redox Forms as New Targets in Duchenne Muscular Dystrophy
IRCCS Ospedale San Raffaele
Unit of Chromatin Dynamics - division of Genetics and Cell Biology
ITALY
LOMBARDIA
MILANO
MYOCITY - A multidimensional single-cell approach to understand muscle dystrophy - IT
IRCCS Ospedale San Raffaele
Istituto di Neurologia Sperimentale - Unità di Ricerca Rigenerazione Neuromuscolare
ITALY
LOMBARDIA
MILANO
An integrated pharmacological/antioxidant approach for Duchenne Muscular Dystrophy: acid sphingomyelinase as new therapeutic target
Università degli Studi di Milano - Biologia e Genetica per le Scienze Mediche
Dipartimento di Scienze Biomediche e Cliniche L. Sacco
ITALY
LOMBARDIA
SAN DONATO MILANESE
Gene editing in Myotonic Dystrophy type 1: assessment of efficiency, safety and therapeutic effect of CTG-repeat deletion in a mouse model of disease
IRCCS Policlinico San Donato
Laboratorio di Cardiologia Molecolare
ITALY
LOMBARDIA
SAN DONATO MILANESE
Circular RNA role in Myotonic Dystrophy type 1
IRCCS Policlinico San Donato
POLICLINICO SAN DONATO
ITALY
TOSCANA
FIRENZE
Towards Precision Medicine with Human Induced Pluripotent Stem Cells for Dystrophin Associated Cardiomyopathy.
Università degli studi di Firenze-Dip. di Medicina Sperimentale e Clinica
Dipartimento di Medicina Sperimentale e Clinica
ITALY
TOSCANA
SESTO FIORENTINO (FIRENZE)
Towards Precision Medicine with Human Induced Pluripotent Stem Cells for Dystrophin Associated Cardiomyopathy.
Università degli studi di Firenze - LENS
European Laboratory for Non-Linear Spectroscopy
ITALY
VENETO
PADOVA
Pre-clinical evaluation of biocompatible nanoparticles as delivery system of 20-methyl-phosphorothioate antisense oligoribonucleotides for exon skippin- mediated dystrophin in restoration
Università degli Studi di Padova- Polo A.Vallisneri
Dipartimento di Istologia, Microbiologia e Biotecnologie Biomediche
ITALY
VENETO
PADOVA
Role of Cyclophilins in Duchenne Muscular Dystrophy
Università degli Studi di Padova- Polo A.Vallisneri
Dipartimento di Scienze Biomediche
ITALY
VENETO
PADOVA
Targeting the Mitochondrial Calcium Uniporter to counteract Duchenne Muscular Dystrophy
Università degli Studi di Padova- Polo A.Vallisneri
Dipartimento di Scienze Biomediche
ITALY
VENETO
TREVISO
Cell therapy for muscle dystrophies: delivery of myogenic precursor cells and amniotic stem cells via polymeric scaffolds
Azienda ULSS 9 - Ospedale di Treviso
Chirurgia Pediatrica
NETHERLANDS
Gelderland
NIJMEGEN
Functional arm training: study for boys with Duchenne Muscular Dystrophy
Radboudumc - Oost
Afdeling Revalidatie
NETHERLANDS
Gelderland
NIJMEGEN
Development of an exoskeleton for the arms for boys with Duchenne Muscular Dystrophy
Radboudumc - Oost
Afdeling Revalidatie
NORWAY
Vestlandet
BERGEN
Betydningen av fysisk trening i oppfølging av barn og unge med Duchenne muskeldystrofi
Haukeland University Hospital
NORWAY
Østlandet
OSLO
Mekanisk hostestøtte for barn med nevromuskulær sykdom og svak hoste
Oslo University Hospital, Ullevaal
Oslo University Hospital, Ullevål
PORTUGAL
CENTRO
ADDRESS: NOT PROVIDED - PT
Understanding the developmental onset of muscular dystrophy in a mouse model of MDC1A.
Institution: Information not provided - PT
SPAIN
Andalucía
CÁDIZ
Cell-type-specific biomarkers in peripheral fluids in trinucleotide diseases
Hospital Universitario Puerta del Mar
Instituto de Investigación e Innovación en Ciencias Biomédicas de Cádiz
SPAIN
Cataluña
BADALONA
EXPLORING HDAC11 FUNCTIONS IN DUCHENNE MUSCULAR DYSTROPHY
IGTP: Institut Germans Trias Pujol
Neuromuscular and Neuropaediatric Research
SPAIN
Cataluña
BARCELONA
New therapies targeting fibroadipogenic cell subpopulations implicated in muscle degeneration
IIB Sant Pau - Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau
Grupo de investigación en enfermedades neuromusculares
SPAIN
Cataluña
BARCELONA
Igenco: In-Depth genomics and cross-omics analysis for undiagnosed rare diseases on a user-friendly collaborative platform
ISGlobal - Instituto de Salud Global de Barcelona
Programa de investigación en Enfermedades no transmisibles y medio ambiente
SPAIN
Cataluña
BARCELONA
ArmTracker: a state-of-the-art wearable system to assess upper limb motor function in real-life conditions for patients with Duchenne muscular dystrophy and spinal muscular atrophy
Instituto de Robótica e Informática Industrial
Grupo de Percepción y Manipulación
SPAIN
Cataluña
BARCELONA
ArmTracker: a state-of-the-art wearable system to assess upper limb motor function in real-life conditions for patients with Duchenne muscular dystrophy and spinal muscular atrophy
Universitat Politècnica de Catalunya
BIO MEC: Laboratorio de Ingeniería Biomecánica
SPAIN
Cataluña
BARCELONA
Nouvelles stratégies pour améliorer la dystrophie musculaire de Duchenne
Universitat Pompeu Fabra. Campus del Mar
Grupo de Biología Celular
SPAIN
Comunidad Valenciana
VALENCIA
ARTHEx biotech: anti-microRNAs for the treatment of myotonic dystrophy
INCLIVA - Facultad de Medicina de la Universidad de Valencia
Grupo de investigación en genómica traslacional
SPAIN
Madrid
MADRID
RAREGenomics: Network of Genomic, Functional, Clinical and Therapeutic Resources for the Study of Rare Neurological Diseases
Hospital Universitario Fundación Jiménez Díaz
Servicio de Genética Clínica
SPAIN
Madrid
MADRID
Functionalized nanoparticles for targeted genome editing in Duchenne Muscular Dystrophy - ES
IMDEA Nano: Madrid Institute of Advanced Studies in Nanoscience
SPAIN
País Vasco
BARAKALDO
Myoblots for rapid evaluation of new treatments for myotonic dystrophy (MaTILDa)
Instituto de Investigación sanitaria Biocruces Bizkaia
Grupo de investigación en enfermedades neuromusculares
SWEDEN
Region Stockholm
SOLNA
Skeletal muscle ribosome specialization in Duchenne muscular dystrophy (DMD) - friend or foe?
Karolinska Institute
SWITZERLAND
Suisse Alémanique
BASEL
MYOCITY - A multidimensional single-cell approach to understand muscle dystrophy - CH
Universität Basel
Biozentrum
SWITZERLAND
Suisse Alémanique
BASEL
Development and Clinical Validation of Magnetic Resonance Methods for the Functional Imaging and Spectroscopy of Skeletal Muscles by Means of Synchronized Electrical Muscle Stimulation
Universitätsspital Basel
Clinic for radiology and nuclear medicine
SWITZERLAND
Suisse Romande
GENÈVE
Duchenne muscular dystrophy: Phenotyping and validation of better murine models for improving preclinical research and clinical translation
UNIGE Université de Genève
Département des sciences pharmaceutiques
UNITED KINGDOM
Northamptonshire
NORTHAMPTON
RNA processing of the brain dystrophin isoform Dp71
University of Northampton
Faculty of Arts, Science and Technology
UNITED KINGDOM
Oxfordshire
OXFORD
MICA: The role of utrophin in DMD and its therapeutic potential
University of Oxford
Department of Physiology, Anatomy and Genetics
UNITED KINGDOM
Oxfordshire
OXFORD
Antisense Oligonucleotide Therapy for Neuromuscular Disease
University of Oxford
Department of Physiology, Anatomy and Genetics
UNITED STATES
California
PALO ALTO
sPAM: Developing peptide-based drugs to target ataxin-2 in neuromuscular disease - US
Stanford University School of Medicine
Department of Genetics
UNITED STATES
Florida
MIAMI
whole body periodic acceleration a novel treatment for duchenne cardiomyopathy in mdx mice
Mount Sinai Medical Center
UNITED STATES
Massachusetts
BOSTON
Discovery of small-molecules modulating new modifier genes in Duchenne Muscular Dystrophy: a novel and promising therapy to escape the dystrophic phenotype
Boston Children's Hospital
UNITED STATES
Massachusetts
BOSTON
Discovery of small-molecules modulating new modifier genes in Duchenne Muscular Dystrophy: a novel and promising therapy to escape the dystrophic phenotype.
Boston Children's Hospital
UNITED STATES
Massachusetts
BOSTON
Toward cell therapy for Duchenne Muscular Dystrophy: characterization of regenerative potential of hIPS-derived Pax7+ cells
Brigham and Women's Hospital
Vertebrate musculo-skeletal development
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
STAT3 signaling network in MuSCs as therapeutic target for DMD
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Use of a novel in vitro DMD model to study muscle fusion during health and disease
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Whole body single aav microgene therapy in canine dmd
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Safety and efficacy of systemic gene therapy in informative models for dmd
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Therapeutics for rare and neglected diseases - science
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
A drug based approach for integrin-mediated alleviation for muscular dystrophy
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Gene therapy platform for rare diseases
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Crispr/cas9-based gene editing for the correction of duchenne muscular dystrophy
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Exploring the role of fbxw7 in regulating satellite cell function and skeletal muscle regeneration
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Innovative approaches to treat duchenne muscular dystrophy using ipsc-derived muscle progenitors
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Interrogating functional and molecular properties of pax7+ putative skeletal muscle stem/progenitor cells derived from human ipscs of healthy donors and duchenne muscular dystrophy patients
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Microtubule regulated mechanotransduction in skeletal muscle
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Mr assessment of bioenergetics and microvascular function in dystrophic muscle
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Multiscale modeling for treatment discovery in duchenne muscular dystrophy
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Muscle tregs in health and disease
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Rna toxicity and muscle regeneration
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Sarcolipin in duchenne muscular dystrophy
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Small molecule approaches to targeting the dna and rna in myotonic dystrophy
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Targeting leukemia inhibitory factor to dystrophic muscle via a macrophage-specific transgene
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Treatment of duchenne muscular dystrophy with the muscle calcium pump
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Assessing and modulating inflammation and fibrosis in dystrophic muscle
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Bioinformatics and genomics
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Highthroughput screening and cell repository
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Optimization of ao drugs 45; 51 & 53
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Biomarker discovery for ao accumulation in kidney
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Clinical evaluation of urine biomarkers for morpholino
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Coupling molecular and clinical phenotypes with ipsc disease modeling for myoediting of duchenne muscular dystrophy
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Correction of muscular dystrophy in mice by crispr/cas9-mediated genomic editing
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Preclinical dosing optimization: dosing schedule; tissue
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Strategies to overcome immunity in gene therapy of dmd
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
The implications of dystrophin-specific t cells for dmd gene correction
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Pediatric toxicity and efficacy in long-term systemic treatment with anti-sense
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
A CRISPR-C2c2 based system to target toxic RNA in myotonic dystrophy type 1
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Functional characterization and therapeutic implications of a dystrophin isoform harboring a deletion in the dystrophin actin binding domain 1
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Prospective Study in Cardiac Disease in Duchenne Muscular Dystrophy (DMD)
Institution: Information not provided - US
CANADA
Colombie-Britannique
VICTORIA
Silent Genomes: Reducing health care disparities and improving diagnostic success for children with genetic diseases from Indigenous populations
University of Victoria
Community Genetics Research Program/Island Medical Program
CANADA
Ontario
TORONTO
Beyond the Genome: Transcriptome Based Diagnostics for Rare Diseases and Cancer
Hospital for Sick Children, Research Institute
FINLAND
Finland
HELSINKI
Mechanisms of RNA toxicity in Myotonic Dystrophy: the role of mitochondrial dysfunction
HiLIFE: Institute of Biotechnology
FRANCE
AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR
Annexins in sarcolemma repair of healthy and pathological human skeletal muscle
Institution: Information not provided - FR
FRANCE
AUVERGNE-RHONE-ALPES
CLERMONT-FERRAND
Rôle des enzymes de conjugaison E2 dans le ciblage dépendant de MuRF1 de la téléthonine dans le muscle squelettique
INRA
Unité de Nutrition Humaine
FRANCE
ILE-DE-FRANCE
EVRY
ReCognitiON - Recognition and Validation of Druggable Targets from the Response to Cognitive Behaviour Therapy in Myotonic Dystrophy type 1 patients from intergrated-Omics Networks - FR (Partner 2)
GENOPOLE - Campus 1
I-Stem - Institut des cellules Souches pour le Traitement et l'Etude des maladies Monogéniques
FRANCE
ILE-DE-FRANCE
EVRY
Consolidating data for rescue of missense mutations from degradation
Généthon
Centre de Recherche Généthon
FRANCE
ILE-DE-FRANCE
PARIS
Role of LRRFIP2 in the control of heart development and skeletal muscle regeneration
Institut Cochin (INSERM U 1016 - CNRS UMR 8104)
Equipe "Développement neuromusculaire, Génétique et Physiopathologie"
FRANCE
ILE-DE-FRANCE
PARIS
Neuroglobin gene overexpression: a promising tool for treating a large spectrum of neurological disorders thanks to its ability to safely and sustainably preserve mitochondrial integrity
Institut National de la Santé et de la Recherche Médicale
FRANCE
ILE-DE-FRANCE
PARIS
ReCognitiON - Recognition and Validation of Druggable Targets from the Response to Cognitive Behaviour Therapy in Myotonic Dystrophy type 1 patients from intergrated - Omics Networks - FR (Partner 1)
Institut de Myologie - Hôpital Pitié-Salpêtrière
Équipe "Dystrophie Myotonique, Physiopathologie & Biothérapie"
FRANCE
OCCITANIE
TOULOUSE
Role of TRIM32, the Limb Girdle Muscular Dystrophy type R8 gene, in neuro-muscular differentiation and homeostasis -FR
CNRS: Centre National de la Recherche Scientifique
GERMANY
Rheinland-Pfalz
COLOGNE
Biomarker discovery and validation for LGMD2I/FKRP-related muscular dystrophy
University of Cologne
Center for Molecular Medicine Cologne
ITALY
FRIULI VENEZIA GIULIA
TRIESTE
Role of TRIM32, the Limb Girdle Muscular Dystrophy type R8 gene, in neuro-muscular differentiation and homeostasis - IT
Università degli Studi di Trieste
Dipartimento di Scienze della Vita
ITALY
LAZIO
ROMA
Diverse rare diseases caused by functional dysregulation of Rho GTPase-related proteins
ISS - Istituto Superiore di Sanità
Centro Nazionale Malattie Rare
ITALY
LIGURIA
GENOVA
Extracellular ATP and T regulatory cells: new therapeutics targets in alpha-sarcoglycan deficient muscular dystrophy (LGMD2D)
IRCCS Istituto G. Gaslini - Ospedale Pediatrico
U.O.C. Neurologia pediatrica e Malattie muscolari
NORWAY
Nord-Norge
TROMSØ
Pathophysiological mechanisms with Limb Girdle muscular dystrophy Type 21
UNN - Universitetssykehuset Nord-Norge
NMK - Nevromuskulært kompetansesenter
NORWAY
Nord-Norge
TROMSØ
Limb-Girdle Muscular Dystrophy in Norway, a cohort study
UNN - Universitetssykehuset Nord-Norge
NMK - Nevromuskulært kompetansesenter
SPAIN
Andalucía
SEVILLA
The involvement of muscle stem cells in the pathology of muscular dystrophy due to POGLUT1 mutations
IBIS - Instituto de Biomedicina de Sevilla
Grupo de enfermedades neuromusculares
SPAIN
Comunidad Valenciana
VALENCIA
Molecular mechanisms in the development of scoliosis in limb-girdle muscular dystrophies
Universitat de València. Facultat de Medicina i Odontologia
Departamento de Bioquímica y Biología Molecular
SPAIN
Madrid
MADRID
Identification of new genes causing rare diseases in pediatric patients, searching of genetic pharmacological targets, and drugs discovery, the steps for a personalized medicine
Instituto de Investigación Hospital 12 de Octubre
Grupo de investigación en enfermedades raras, mitocondriales y neuromusculares
UNITED KINGDOM
Tyne & Wear
NEWCASTLE-UPON-TYNE
Defining Clinical Endpoints in Limb Girdle Muscular Dystrophy (LGMD) (GRASP)
Newcastle University
Newcastle Clinical Trials Unit
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
A powerful web-based discovery platform for rare disease genetics
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Chaperone dysfunction in myopathy: connecting yeast genetics with mouse models
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Mechanosignaling functions of the dystrophin glycoprotein complex in muscular dystrophy
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Targeting dystroglycanopathies using pluripotent-derived myogenic progenitors
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
The sarcoglycan complex in skeletal muscle mechanotransduction
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
The role of dystroglycan in neural circuit development.
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Functional mechanisms underlying dystroglycan-dependent and independent roles of protein o-mannosylation in the nervous system
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Membrane remodeling in viral infection; parasite invasion; apoptosis; and cancer
Institution: Information not provided - US
Multicentric Research projects
- Osaka University Graduate School of Medicine
- TAYS - Tampere University Hospital
- Neuromuscular Center
- Université Pierre et Marie Curie - Paris 6
- Groupe Myologie - UMR S 787
- Institut für Medizinische Genetik und angewandte Genomik Tübingen
- Institut für Medizinische Genetik und angewandte Genomik
- Institut de génétique et de biologie moléculaire et cellulaire - IGBMC
- Département Médecine translationnelle et neurogénétique
- Institut de Génétique Humaine (IGH)
- Département Génétique et Développement
- A.O.U. Sant'Anna - Polo Chimico-Bio-Medico
- U.O.C. di Genetica Medica
- Sapienza Università di Roma
- Dipartimento di Biologia e Biotecnologie "Charles Darwin"
- IRCCS Ospedale San Raffaele
- Fondazione Centro San Raffaele del Monte Tabor - Divisione di Medicina Rigenerativa Cellule Staminali e Terapia Genica (DMR)
- IRCCS Ospedale San Raffaele
- Fondazione Centro San Raffaele del Monte Tabor - Divisione di Medicina Rigenerativa Cellule Staminali e Terapia Genica (DMR)
- Policlinico Universitario di Padova
- Dipartimento di Medicina Molecolare
- Ottawa Hospital Research Institute
- Sprott Center for Stem Cell Research
- Institution: Information not provided - FR
- Faculté de médecine de Clermont-Ferrand
- Laboratoire de Génétique, Reproduction et Développement
- Institut Cochin (INSERM U 1016 - CNRS UMR 8104)
- Equipe "Développement neuromusculaire, Génétique et Physiopathologie"
- Institut de Myologie - Hôpital Pitié-Salpêtrière
- Équipe "Dystrophie Myotonique, Physiopathologie & Biothérapie"
- CHU de Marseille - Hôpital de la Timone
- Laboratoire de génétique moléculaire
- Zentrum für Kinder- und Jugendmedizin Freiburg
- Klinik für Neuropädiatrie und Muskelerkrankungen
- Charité / Max-Delbrück-Centrum für Molekulare Medizin
- Muskelforschung mit Hochschulambulanz für Muskelkrankheiten
- University of Tel-Aviv
- Dep. of Bio-Medical Engineering
- Policlinico S. Orsola-Malpighi - Area S. Orsola
- Università di Bologna
- Consiglio Nazionale delle Ricerche
- CNR
- IRCCS Fondazione Santa Lucia
- Laboratorio di Epigenetica e Farmacologia Rigenerativa
- IRCCS Fondazione Santa Lucia
- Laboratorio di Epigenetica e Trasduzione del Segnale
- Sapienza Università di Roma
- FISIOLOGIA E FARMACOLOGIA "VITTORIO ERSPAMER"
- IRCCS Ospedale San Raffaele
- Istituto di Ricerca per le Cellule Staminali
- Università degli Studi di Bari Aldo Moro
- Sezione di Farmacologia
- University of Trento
- Dulbecco Telethon Laboratory of Stem Cells and Regenerative Medicine
- Azienda Ospedaliera di Padova
- Clinica Pediatrica
- Radboudumc - Radboud universitair medisch centrum
- Afdeling Neurologie
- Institution: Information not provided - NL
- Universitäts-Kinderspital beider Basel UKBB
- Die Abteilung für Neuro- und Entwicklungspädiatrie
- Newcastle upon Tyne Hospitals NHS Trust
- John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine
- Newcastle upon Tyne Hospitals NHS Trust
- John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine
- Stanford University School of Medicine
- Department of Genetics
- Università degli Studi di Trieste
- Dipartimento di Scienze della Vita
- Radboudumc - Radboud universitair medisch centrum
- Centrum voor Moleculaire en Biomoleculaire Informatica
- Haukeland University Hospital
JAPAN
JAPAN
OSAKA
Identification of factors inducing MuSC expansion from overloaded muscle
FINLAND
Finland
TAMPERE
IDOLS-G: Improved diagnostic output in large sarcomeric genes
FRANCE
ILE-DE-FRANCE
PARIS
ENDOSTEM: Activation of vasculature associated stem cells and muscle stem cells for the repair and maintenance of muscle tissue
GERMANY
Baden-Württemberg
TÜBINGEN
Neuromics: Integrated European -omics research project for diagnosis and therapy in rare neuromuscular and neurodegenerative diseases
FRANCE
GRAND-EST
ILLKIRCH-GRAFFENSTADEN
HEART DM: Exploring the mechanisms of heart dysfunctions in myotonic dystrophies
FRANCE
OCCITANIE
MONTPELLIER
riboOPMD: Ribosomal and small non-coding RNAs in oculopharyngeal muscular dystrophy
ITALY
EMILIA ROMAGNA
FERRARA
BIO-NMD: identifying and validating pre-clinical biomarkers for diagnostics and therapeutics of neuromuscular disorders
ITALY
LAZIO
ROMA
Functional analysis of separase-dependent lamins' regulation in AD-EDMD
ITALY
LOMBARDIA
MILANO
inter-FSHD-epigen: An international effort to understand FSHD muscular dystrophy epigenetics
ITALY
LOMBARDIA
MILANO
EpiThe4FSHD: Safety and efficacy of a possible epigenetic therapy for FSHD muscular dystrophy
ITALY
VENETO
PADOVA
The neuromuscular junction as a novel therapeutic target for Collagen VI myopathies
CANADA
Ontario
OTTAWA
SIRD: Stimulating Intrinsic Repair for DMD
FRANCE
AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR
Duchenne Muscular Dystrophy non-viral gene therapy
FRANCE
AUVERGNE-RHONE-ALPES
CLERMONT-FERRAND
MYORES: European Muscle Development Network (FINISHED)
FRANCE
ILE-DE-FRANCE
PARIS
MYOCITY: A multidimensional single-cell approach to understand muscle dystrophy
FRANCE
ILE-DE-FRANCE
PARIS
Pre-clinical development of peptide-oligonucleotides for DM1
FRANCE
PROVENCE-ALPES-COTE D'AZUR
MARSEILLE
NMD-CHIP: development of targeted DNA-chips for high throughput diagnosis of neuromuscular disorders
GERMANY
Baden-Württemberg
FREIBURG
CARE-NMD : Improving care for Duchenne muscular dystrophy
GERMANY
Berlin
BERLIN
EUROMUSCLENET: Europäisches Informations-Netzwerk über muskuläre Erkrankungen
ISRAEL
ISRAEL
TEL AVIV
A quantitative MRI platform for simultaneous and automatic quantification of fat infiltration and T2 relaxation times in neuromuscular disorders
ITALY
EMILIA ROMAGNA
BOLOGNA
Morphofunctional characterization of transportin 3 (TNPO3) in the pathogenesis of limb-girdle muscular dystrophy 1F
ITALY
LAZIO
ROMA
Deciphering the role of Prdm16-mediated H3K9 methylation in the control of Fibro-Adipogenic Progenitors identity and skeletal muscle repair
ITALY
LAZIO
ROMA
Identification of the chromatin profile predicting the responsiveness of dystrophic patients to epigenetic therapy
ITALY
LAZIO
ROMA
Functionalized nanoparticles for targeted genome editing in Duchenne Muscular Dystrophy
ITALY
LAZIO
ROMA
Role of glia in neurological comorbidities of Duchenne muscular dystrophy
ITALY
LOMBARDIA
MILANO
OptiStem: Ottimizzazione della terapia con cellule staminali nelle sperimentazioni cliniche per le malattie muscolari cutanee e degenerative
ITALY
PUGLIA
BARI
Selected growth hormone secretagogues (GHS) with wide mechanism of actions as disease modifiers in Duchenne Muscular Dystrophy: a multidisciplinary proof-of-concept study in mdx mouse model
ITALY
TRENTINO ALTO ADIGE
POVO
Novel satellite cell heterogeneity in healthy and pathological regeneration
ITALY
VENETO
PADOVA
Brains for brain: research group of the European task force on brain and neurodegenerative lysosomal storage diseases
NETHERLANDS
Gelderland
NIJMEGEN
OPTIMISTIC: Observational Prolonged Trial In Myotonic dystrophy type 1 to Improve QoL-Standards, a Target Identification Collaboration
NETHERLANDS
Utrecht
ADDRESS: NOT PROVIDED - NL
ReCognitION: Recognition and Validation of Druggable Targets from the Response to Cognitive Behaviour Therapy in Myotonic Dystrophy type 1 patients from Integrated -Omics Networks
SWITZERLAND
Suisse Alémanique
BASEL
TAMDMD : Tamoxifen in Duchenne muscular dystrophy - a randomised placebo controlled phase 2 trial
UNITED KINGDOM
Tyne & Wear
NEWCASTLE UPON TYNE
TREAT-NMD: Accelerating Treatments for Neuromuscular Diseases (research)
UNITED KINGDOM
Tyne & Wear
NEWCASTLE UPON TYNE
BIOIMAGE-Neuromuscular Diseases
UNITED STATES
California
PALO ALTO
sPAM: Developing peptide-based drugs to target ataxin-2 in neuromuscular disease
ITALY
FRIULI VENEZIA GIULIA
TRIESTE
Role of TRIM32, the Limb Girdle Muscular Dystrophy type R8 gene, in neuro-muscular differentiation and homeostasis
NETHERLANDS
Gelderland
NIJMEGEN
ReCognitiON - Recognition and Validation of Druggable Targets from the Response to Cognitive Behaviour Therapy in Myotonic Dystrophy type 1 patients from integrated-Omics Networks
NORWAY
Vestlandet
BERGEN