Search for a research project
Other search option(s)
237 Result(s)
Terminated research projects = 
Funded by an IRDiRC member = 
Member of a ERN = 
Research projects
CANADA
Ontario
HAMILTON
Role of Xin, an actin-binding protein, in satellite cells and muscular dystrophies
McMaster University
Department of Pathology and Molecular Medicine
FRANCE
HAUTS-DE-FRANCE
ADDRESS: NOT PROVIDED - FR
Characterization of Liver-induced allo-specific CD8 regulatory T lymphocytes
Institution: Information not provided - FR
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
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
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
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Interventions in genetic counseling
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
Engineering Clinical Trials on a Chip for Dystrophin-Deficient Muscular Dystrophy
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Project 2 - Duchenne muscular distrophy
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
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
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
Elucidation of Wnt7a mechanism of action for muscle regeneration
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
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
CANADA
Québec
QUÉBEC
Multifunctional properties of osteoprotegerin in muscular dystrophy and muscle repair
CHUQ - (CHUL) Centre hospitalier de l'Université Laval
Axe Neuosciences, CHUL
FRANCE
HAUTS-DE-FRANCE
ADDRESS: NOT PROVIDED - FR
Gene transfer tolerance in combined liver and muscle rAAV gene therapy
Institution: Information not provided - FR
FRANCE
HAUTS-DE-FRANCE
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
HAUTS-DE-FRANCE
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
HAUTS-DE-FRANCE
ADDRESS: NOT PROVIDED - FR
Dystrophin-deficient diaphragm dysfunction induced by mechanical ventilation in mdx mice.
Institution: Information not provided - FR
FRANCE
HAUTS-DE-FRANCE
ADDRESS: NOT PROVIDED - FR
SIX homeoproteins and muscle stem cells properties
Institution: Information not provided - FR
FRANCE
HAUTS-DE-FRANCE
ADDRESS: NOT PROVIDED - FR
Liver Gene Transfer-Induced allogeneic Tolerance for organ transplantation and cell therapy
Institution: Information not provided - FR
FRANCE
HAUTS-DE-FRANCE
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
HAUTS-DE-FRANCE
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
HAUTS-DE-FRANCE
ADDRESS: NOT PROVIDED - FR
Elucidate the cellular and molecular mechanisms involved in the generation of revertant dystrophin-positive fibers using the dystrophic DmdEGFP-mdx reporter mouse
Institution: Information not provided - FR
FRANCE
HAUTS-DE-FRANCE
ADDRESS: NOT PROVIDED - FR
Involvement of endo/sarcoplasmic reticulum - mitochondria interaction in the Duchenne muscular dystrophy
Institution: Information not provided - FR
FRANCE
HAUTS-DE-FRANCE
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
HAUTS-DE-FRANCE
ADDRESS: NOT PROVIDED - FR
Duchenne Muscular Dystrophy non-viral gene therapy - FR
Institution: Information not provided - FR
FRANCE
ILE-DE-FRANCE
CORBEIL-ESSONNES
GMP production for rare diseases
YposKesi (AFM-Telethon-Genethon)
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
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
Consequence of senescence in muscle regeneration and degenerative myopathies
Institut Pasteur
Cellular plasticity in age-related pathologies
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
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
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
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
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
Baden-Württemberg
FREIBURG
GrowDMD - Growing up with Duchenne Muscular Dystrophy -DE
Zentrum für Kinder- und Jugendmedizin Freiburg
Klinik für Neuropädiatrie und Muskelerkrankungen
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
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
Understanding the contribution of molecular mediators of inflammation to DMD progression and implications for therapy: studying the impact of IL-6 transignaling on dystrophic muscle stability
Sapienza Università di Roma - Sezione di Ricerca "Valdoni Lab"
Dipartimento di Istologia ed Embriologia Medica
ITALY
LAZIO
ROMA
Unveiling the cytoplasmic functions of HDAC4 in dystrophic skeletal muscle
Sapienza Università di Roma - Sezione di Ricerca "Valdoni Lab"
Dipartimento di Scienze Anatomiche, Istologiche, Medico Legali e dell'Apparato Locomotore
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
Towards Precision Medicine with Human Induced Pluripotent Stem Cells for Dystrophin Associated Cardiomyopathy.
Centro Cardiologico Monzino
Unità di Biologia vascolare e Medicina rigenerativa
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
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
TRENTINO ALTO ADIGE
POVO
Novel satellite cell heterogeneity in healthy and pathological regeneration - IT
University of Trento
Dulbecco Telethon Laboratory of Stem Cells and Regenerative Medicine
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
PADOVA
Identification of novel druggable targets for 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
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
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
Cataluña
ESPLUGUES DE LLOBREGAT
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
Hospital Sant Joan de Déu Barcelona
Unidad de Enfermedades Neuromusculares
SWEDEN
Region Stockholm
SOLNA
Skeletal muscle ribosome specialization in Duchenne muscular dystrophy (DMD) - friend or foe?
Karolinska Institutet
Karolinska Institute
SWITZERLAND
Suisse Alémanique
BASEL
MYOCITY - A multidimensional single-cell approach to understand muscle dystrophy - CH
Universität Basel
Biozentrum
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
Florida
MIAMI
whole body periodic acceleration a novel treatment for duchenne cardiomyopathy in mdx mice
Mount Sinai Medical Center
UNITED STATES
Maryland
BETHESDA
Gene therapy platform for rare diseases
National Center for Advancing Translational Sciences (NCATS-NIH)
Therapeutic Development Branch
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
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
Sarcolipin in duchenne muscular 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
Translational and pre-clinical studies of aav-mediated gene therapy of muscular
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
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
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Molecular mechanisms of telomere function in muscle stem cells
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Structure-Function Analysis of Sarcospan
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Altered nucleus-cytoskeleton coupling in dystrophic muscle
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Magnetic Resonance Imaging and Biomarkers for Muscular Dystrophy
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Alternative delivery of adeno-associated virus therapeutic vector for the treatment of neuromuscular disorders
Institution: Information not provided - US
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
Project 1 - Duchenne muscular distrophy
Institution: Information not provided - US
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
AUVERGNE-RHONE-ALPES
VILLEURBANNE
Muscular dystrophies: the role of the extracellular matrix
Université Claude Bernard Lyon 1
FRANCE
HAUTS-DE-FRANCE
ADDRESS: NOT PROVIDED - FR
Pro-inflammatory Cytokines as Potential Therapeutic Target in Type 1 Facioscapulohumeral Muscular Dystrophy: Pilot Study
Institution: Information not provided - FR
FRANCE
HAUTS-DE-FRANCE
ADDRESS: NOT PROVIDED - FR
Identification of factors inducing MuSC expansion from overloaded muscle - FR
Institution: Information not provided - FR
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
FRANCE
PROVENCE-ALPES-COTE D'AZUR
AIX-EN-PROVENCE
The search for genetic clues in muscular dystrophy
Aix-Marseille Université
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
UNITED STATES
Washington
ADDRESS: NOT PROVIDED - US
In vivo role of the fibroblast in muscular dystrophy
Institution: Information not provided - US
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
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
CANADA
Ontario
OTTAWA, ONTARIO
Inhibition of HDAC6 as Therapeutic Strategy in Neuromuscular Diseases
University of Ottawa
Faculty of Medicine -
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
HAUTS-DE-FRANCE
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
HAUTS-DE-FRANCE
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
HAUTS-DE-FRANCE
ADDRESS: NOT PROVIDED - FR
Impact of blowpipe training on cough efficiency in neuromuscular patients : a prospective evaluation.
Institution: Information not provided - FR
FRANCE
HAUTS-DE-FRANCE
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
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
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
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
GARCHES
Early identification of respiratory exacerbations using NIV device monitoring in slowly progressive neuromuscular disorders
CHU Paris IdF Ouest - Hôpital Raymond Poincaré
Service de Physiologie-Explorations Fonctionnelles - Consultations Troubles du sommeil
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
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
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
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
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
LAZIO
ROMA
Cellular network driving neuromuscular junction stability
Sapienza Università di Roma
Università degli Studi di Roma "La Sapienza"
ITALY
LAZIO
ROMA
Unraveling HSPB3 physiological functions to understand its implication in neuromuscular diseases
Sapienza Università di Roma - Dip. di Biologia e Biotecnologie Charles Darwin
Dipartimento di Biologia e Biotecnologie - Charles Darwin
NETHERLANDS
Zuid-Holland
LEIDEN
Understanding histopathological alterations in neuromuscular disorders by spatial transcriptomics
LUMC - Leids Universitair Medisch Centrum
Afdeling Humane Genetica
NORWAY
Østlandet
OSLO
Mekanisk hostestøtte for barn med nevromuskulær sykdom og svak hoste
Oslo University Hospital, Ullevaal
Oslo University Hospital, Ullevål
SPAIN
Cataluña
BARCELONA
Igenco: In-Depth genomics and cross-omics analysis for undiagnosed rare diseases on a user-friendly collaborative platform
Centro de Regulación Genómica
Centro Nacional de Análisis Genómico
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
UNITED KINGDOM
Tyne & Wear
NEWCASTLE UPON TYNE
Implementation of an artificial intelligence module on the web-based digital platform MyoShare for guiding the diagnosis of muscle diseases.
University of Newcastle
John Walton Muscular Dystrophy Research Centre
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
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
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
ITALY
LAZIO
ROMA
Diverse rare diseases caused by functional dysregulation of Rho GTPase-related proteins
ISS - Istituto Superiore di Sanità
Centro Nazionale Malattie Rare
SPAIN
Andalucía
SEVILLA
Development of bioinformatic tools for the automatic handling of secondary findings and their use in genetic and pharmacogenetic counseling
Fundación Progreso y Salud
Área de bioinformática clínica
SPAIN
Cataluña
BARCELONA
Catalan Interhospital Network of Genetic Variants to improve genetic diagnosis in rare diseases
Centro de Regulación Genómica
Archivo europeo de genomas y fenomas del CRG
SPAIN
Cataluña
BARCELONA
Catalan Interhospital Network of Genetic Variants to improve genetic diagnosis in rare diseases
Hospital Clínic de Barcelona
Servicio de Inmunologia
SPAIN
Cataluña
BARCELONA
Catalan Interhospital Network of Genetic Variants to improve genetic diagnosis in rare diseases
Hospital Universitari Vall d'Hebron
Laboratorio de Genética
SPAIN
Cataluña
BARCELONA
Catalan Interhospital Network of Genetic Variants to improve genetic diagnosis in rare diseases
Hospital de la Santa Creu i Sant Pau
Servicio de Genética
SPAIN
Cataluña
ESPLUGUES DE LLOBREGAT
Federated network for functional genomics of undiagnosed and rare diseases, RareFunction
Fundació Sant Joan de Déu - Institut de Recerca Sant Joan de Déu
Grupo de investigación en Neurogenética y Medicina Molecular
SPAIN
Cataluña
ESPLUGUES DE LLOBREGAT
Catalan Interhospital Network of Genetic Variants to improve genetic diagnosis in rare diseases
Hospital Sant Joan de Déu Barcelona
Servicio de Laboratorio
SPAIN
Cataluña
L'HOSPITALET DE LLOBREGAT
Catalan Interhospital Network of Genetic Variants to improve genetic diagnosis in rare diseases
Hospital Universitari de Bellvitge
Unidad de Genética 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
Multicentric Research projects
- Ottawa Hospital Research Institute
- Sprott Center for Stem Cell Research
- Faculté de médecine de Clermont-Ferrand
- Laboratoire de Génétique, Reproduction et Développement
- Institution: Information not provided - FR
- Institut Cochin (INSERM U 1016 - CNRS UMR 8104)
- Equipe "Développement neuromusculaire, Génétique et Physiopathologie"
- Université Pierre et Marie Curie - Paris 6
- Groupe Myologie - UMR S 787
- Zentrum für Kinder- und Jugendmedizin Freiburg
- Klinik für Neuropädiatrie und Muskelerkrankungen
- A.O.U. Sant'Anna - Polo Chimico-Bio-Medico
- U.O.C. di Genetica Medica
- 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
- University of Trento
- Dulbecco Telethon Laboratory of Stem Cells and Regenerative Medicine
- 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
- Institut für Medizinische Genetik und angewandte Genomik Tübingen
- Institut für Medizinische Genetik und angewandte Genomik
- Osaka University Graduate School of Medicine
- TAYS - Tampere University Hospital
- Neuromuscular Center
- Charité / Max-Delbrück-Centrum für Molekulare Medizin
- Muskelforschung mit Hochschulambulanz für Muskelkrankheiten
- University of Tel-Aviv
- Dep. of Bio-Medical Engineering
- Azienda Ospedaliera di Padova
- Clinica Pediatrica
- Stanford University School of Medicine
- Department of Genetics
CANADA
Ontario
OTTAWA
SIRD: Stimulating Intrinsic Repair for DMD
FRANCE
AUVERGNE-RHONE-ALPES
CLERMONT-FERRAND
MYORES: European Muscle Development Network (FINISHED)
FRANCE
HAUTS-DE-FRANCE
ADDRESS: NOT PROVIDED - FR
Duchenne Muscular Dystrophy non-viral gene therapy
FRANCE
ILE-DE-FRANCE
PARIS
MYOCITY: A multidimensional single-cell approach to understand muscle dystrophy
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
FREIBURG
CARE-NMD : Improving care for Duchenne muscular dystrophy
ITALY
EMILIA ROMAGNA
FERRARA
BIO-NMD: identifying and validating pre-clinical biomarkers for diagnostics and therapeutics of neuromuscular disorders
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
TRENTINO ALTO ADIGE
POVO
Novel satellite cell heterogeneity in healthy and pathological regeneration
SWITZERLAND
Suisse Alémanique
BASEL
TAMDMD : Tamoxifen in Duchenne muscular dystrophy - a randomised placebo controlled phase 2 trial
UNITED KINGDOM
Tyne & Wear
NEWCASTLE UPON TYNE
BIOIMAGE-Neuromuscular Diseases
GERMANY
Baden-Württemberg
TÜBINGEN
Neuromics: Integrated European -omics research project for diagnosis and therapy in rare neuromuscular and neurodegenerative diseases
JAPAN
JAPAN
OSAKA
Identification of factors inducing MuSC expansion from overloaded muscle
FINLAND
Finland
TAMPERE
IDOLS-G: Improved diagnostic output in large sarcomeric genes
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
VENETO
PADOVA
Brains for brain: research group of the European task force on brain and neurodegenerative lysosomal storage diseases
UNITED STATES
California
PALO ALTO