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CANADA

Alberta
CALGARY

Finanziato da un ente associato a IRDiRCIdentifying Cellular and Molecular Modifiers of Muscular Dystrophy
University of Calgary
Department of Biochemistry and Molecular Biology

CANADA

Ontario
OTTAWA

Finanziato da un ente associato a IRDiRCDifferential role of myogenic regulatory factors in establishing muscle-specific gene expression
Ottawa Hospital Research Institute
Sprott Center for Stem Cell Research

CANADA

Ontario
OTTAWA

Finanziato da un ente associato a IRDiRCSatellite 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

Finanziato da un ente associato a IRDiRCSIX family transcription factors in adult muscle regeneration.
University of Ottawa
Faculty of Medicine -

FRANCIA

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCIA

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCIA

ILE-DE-FRANCE
EVRY

Finanziato da un ente associato a IRDiRCWhite adipose tissue: a stem/stromal cell provider involved in muscle regeneration?
AFM Téléthon - Association Française contre les Myopathies

FRANCIA

OCCITANIE
TOULOUSE

Finanziato da un ente associato a IRDiRCIntermuscular adipose tissue as a trigger of muscle wasting?
Institut des Maladies Métaboliques et Cardiovasculaires
Laboratoire de recherche sur les obésités

ITALIA

EMILIA ROMAGNA
ADDRESS: NOT PROVIDED - IT

ITALIA

LAZIO
ROMA

Finanziato da un ente associato a IRDiRCCaratterizzazione del pathway di regolazione cellulare e trascrizione muscolo specifica: implicazioni terapeutiche per la rigenerazione muscolare
Parco Scientifico Biomedico di Roma San Raffaele
Laboratorio di Endocrinologia e Metabolismo Molecolare

GIAPPONE

JAPAN
TOKYO

Finanziato da un ente associato a IRDiRCEstablishment of Integrated Genome Analysis Center for Hereditary Muscle Disease
National Center of Neurology and Psychiatry
Department of Neuromuscular Research

AUSTRIA

WIEN
WIEN

Molecular mechanisms of plectin-related muscular dystrophy
Medizinische Universität Wien
Zentrum für Anatomie und Zellbiologie

CANADA

Ontario
HAMILTON

Finanziato da un ente associato a IRDiRCRole of Xin, an actin-binding protein, in satellite cells and muscular dystrophies
McMaster University
Department of Pathology and Molecular Medicine

FINLANDIA

Finland
HELSINKI

FRANCIA

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCIA

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

Finanziato da un ente associato a IRDiRCBone morphogenetic proteins regulate patterning of limb muscles.
Institution: Information not provided - FR

FRANCIA

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCIA

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCIA

BRETAGNE
RENNES

Structure and function of dystrophin in relation with mutations / deletions in the human dystrophinopathies
Faculté de médecine de Rennes
Institut de Génétique et Développement de Rennes - UMR6290

FRANCIA

GRAND-EST
ILLKIRCH-GRAFFENSTADEN

Finanziato da un ente associato a IRDiRCPhysiopathology of muscle atrophy in myotonic dystrophy
Institut de génétique et de biologie moléculaire et cellulaire - IGBMC
Département Médecine translationnelle et neurogénétique

FRANCIA

ILE-DE-FRANCE
CRÉTEIL

Finanziato da un ente associato a IRDiRCiDM-scope: the international French-Quebec myotonic dystrophy registry
CHU Henri Mondor
Centre expert de maladie neuro-musculaire

FRANCIA

ILE-DE-FRANCE
EVRY

Finanziato da un ente associato a IRDiRCFAT1 Signaling in skeletal muscle growth and repair: Muscle versus mesenchyme perspectives
AFM Téléthon - Association Française contre les Myopathies

FRANCIA

ILE-DE-FRANCE
FONTENAY-AUX-ROSES

Finanziato da un ente associato a IRDiRCFunctional analysis of separase-dependent lamins' regulation in AD-EDMD
Institut de biologie François Jacob
Laboratoire Réparation Et Vieillissement - LREV

FRANCIA

ILE-DE-FRANCE
PARIS

Finanziato da un ente associato a IRDiRCASC-1, a novel actor in the pathophysiology of congenital muscle disorders
Institut de Myologie - Hôpital Pitié-Salpêtrière
Unité clinique de pathologie neuromusculaire

FRANCIA

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"

FRANCIA

OCCITANIE
MONTPELLIER

Effects of antioxidant supplementation in patients (Facio-scapulo-humeral dystrophy)
CHU de Montpellier - Hôpital Arnaud de Villeneuve
Département de Physiologie Clinique - Exploration métabolique

FRANCIA

OCCITANIE
MONTPELLIER

New therapeutic strategies development for oculopahryngeal muscular dystrophy using a drosophila model
Institut de Génétique Humaine (IGH)
Département Génétique et Développement

FRANCIA

OCCITANIE
MONTPELLIER

Finanziato da un ente associato a IRDiRCriboOPMD: Ribosomal and small non-coding RNAs in oculopharyngeal muscular dystrophy - FR
Institut de Génétique Humaine (IGH)
Département Génétique et Développement

FRANCIA

PROVENCE-ALPES-COTE D'AZUR
MARSEILLE

Finanziato da un ente associato a IRDiRCCharacterization 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

FRANCIA

PROVENCE-ALPES-COTE D'AZUR
MARSEILLE

Finanziato da un ente associato a IRDiRCFAT1 Signaling in skeletal muscle growth and repair: Muscle versus mesenchyme perspectives
IBDM - Institut de Biologie du Développement de Marseille
Développement et pathologies des circuits neuromusculaires

FRANCIA

PROVENCE-ALPES-COTE D'AZUR
NICE

Finanziato da un ente associato a IRDiRCFrench National Registry for FacioScapuloHumeral muscular Dystrophy (FSHD)
CHU de Nice - Hôpital Pasteur
Service Neurologie - Système Nerveux Périphérique et Muscles / Sclérose Latérale Amyotrophique

FRANCIA

PROVENCE-ALPES-COTE D'AZUR
NICE

Finanziato da un ente associato a IRDiRCResolve 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

FRANCIA

PROVENCE-ALPES-COTE D'AZUR
NICE

Immuno-histochemical and molecular diagnosis of epidermolysis bullosa (COL17A1, PLEC1, LAMA3, LAMB3, LAMC2, COL7A1, ITGB4 and ITGA6 genes)
Faculté de médecine de Nice Sophia-Antipolis
Biologie et physiopathologie cutanée : expression génique, signalisation et thérapie

FRANCIA

PROVENCE-ALPES-COTE D'AZUR
NICE

Finanziato da un ente associato a IRDiRCDeciphering the role of the heterochromatin conformation in Emery Dreifuss Muscular Dystrophy (EDMD)
Institute for Research on Cancer and Aging
Department of Medical Genetics

GIAPPONE

JAPAN
AICHI

ITALIA

EMILIA ROMAGNA
ADDRESS: NOT PROVIDED - IT

ITALIA

LAZIO
ROMA

Finanziato da un ente associato a IRDiRCLo studio RAMYD (valutazione del rischio aritimico nella Distrofia Miotonica tipo I): la fase 2
Università Cattolica del Sacro Cuore
Istituto di Anatomia Umana e Biologia Cellulare

ITALIA

LOMBARDIA
MILANO

ITALIA

LOMBARDIA
MILANO

Finanziato da un ente associato a IRDiRCUn modello animale per sviluppare strategie terapeutiche per la distrofia muscolare facio-scapolo-omerale (completato)
IRCCS Ospedale San Raffaele
Fondazione Centro San Raffaele del Monte Tabor - Divisione di Medicina Rigenerativa Cellule Staminali e Terapia Genica (DMR)

ITALIA

LOMBARDIA
MILANO

Finanziato da un ente associato a IRDiRCModulazione dell'espressione della proteina p27 per migliorare la Distrofia Muscolare Congenita da deficit di Merosina (MDC1A)
IRCCS Ospedale San Raffaele
Istituto di Neurologia Sperimentale - Unità di Ricerca Rigenerazione Neuromuscolare

ITALIA

LOMBARDIA
MILANO

Finanziato da un ente associato a IRDiRCAnalisi dei meccanismi epigenetici responsabili dell'insorgenza della Distrofia Facioscapolomerale (completato)
Università degli Studi di Milano - Biologia e Genetica per le Scienze Mediche
Dipartimento di Biologia e Genetica per le Scienze Mediche

ITALIA

SICILIA
TROINA

PAESI BASSI

Zuid-Holland
LEIDEN

Finanziato da un ente associato a IRDiRCRNA processing role in muscle degeneration opens therapeutical options for adult myopathies
LUMC - Leids Universitair Medisch Centrum
Afdeling Humane Genetica

REGNO UNITO; GRAN BRETAGNA

Greater London
LONDON

Finanziato da un ente associato a IRDiRCUnderstanding and Ameliorating Pathogenesis in FSHD
School of Biomedical sciences - King's College London
Randall Division of Cell and Molecular Biophysics

REGNO UNITO; GRAN BRETAGNA

Greater London
LONDON

Finanziato da un ente associato a IRDiRCModelling FSHD as a tool for testing therapeutics
School of Biomedical sciences - King's College London
Randall Division of Cell and Molecular Biophysics

REGNO UNITO; GRAN BRETAGNA

Oxfordshire
OXFORD

Finanziato da un ente associato a IRDiRCAdvanced peptide-oligonucleotide therapy for Myotonic Dystrophy Type 1
University of Oxford
Department of Physiology, Anatomy and Genetics

SPAGNA

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

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

Finanziato da un ente associato a IRDiRCInterventions in genetic counseling
Institution: Information not provided - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

Finanziato da un ente associato a IRDiRCGenetic and epigenetic mechamisms of fshd pathogenesis
Institution: Information not provided - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

Finanziato da un ente associato a IRDiRCMuscle-specific regulation of pabpn1 expression
Institution: Information not provided - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

Finanziato da un ente associato a IRDiRCMyonuclear homeostasis in craniofacial muscles
Institution: Information not provided - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

Finanziato da un ente associato a IRDiRCRegulation of subcellular organization in skeletal muscle
Institution: Information not provided - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

Finanziato da un ente associato a IRDiRCMechanisms of cognitive deficits in dystroglycanopathies
Institution: Information not provided - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

Finanziato da un ente associato a IRDiRCGenetic modifiers of fshd
Institution: Information not provided - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

Finanziato da un ente associato a IRDiRCAnimal models of fshd for therapy
Institution: Information not provided - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

Finanziato da un ente associato a IRDiRCBiomarker discovery in muscles from fshd patients
Institution: Information not provided - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

Finanziato da un ente associato a IRDiRCFshd disease biomarkers
Institution: Information not provided - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

Finanziato da un ente associato a IRDiRCMyogenesis studies for fshd biomarkers
Institution: Information not provided - US

SVEZIA

Region Skåne
LUND

Laminins and Cib2 in congenital muscular dystrophy
Lund University
Department of Experimental Medical Science

SVEZIA

Region Stockholm
HUDDINGE

The functional organisation of the brain
Karolinska Institutet - Huddinge
Department of Clinical Neuroscience

SVEZIA

Region Stockholm
STOCKHOLM

Genetic and molecular mechanisms in familial visual impairment
Karolinska Institutet - Solna
Department of molecular medicine and surgery

AUSTRIA

WIEN
WIEN

Finanziato da un ente associato a IRDiRCLong-Term Observational Study of Translarna Safety and Effectiveness in Usual Care - AT
Gottfried von Preyer'sches Kinderspital
Abteilung für Kinder- und Jugendheilkunde

AUSTRIA

WIEN
WIEN

Cardiac calcium channel abnormalities in Duchenne Muscular Dystrophy
Medizinische Universität Wien
Zentrum für Physiologie und Pharmakologie

BELGIO

ANTWERPEN
ANTWERPEN

BELGIO

ARRONDISSEMENT BRUSSELS-CAPITAL
BRUSSELS

Finanziato da un ente associato a IRDiRCNEXTGEN-AAV: Development of next-generation AAV vectors for Duchenne muscular dystrophy
VUB_ Vrije Universiteit Brussel, Campus Jette
Department of Gene Therapy and Regenerative Medicine

BELGIO

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

BELGIO

VLAAMS BRABANT
HEVERLEE (LEUVEN)

"3DMyoDM1": A novel human 3D in-vitro disease model for DM1
Laboratory of Bioengineering and Morphogenesis, KU Leuven
Laboratory of Bioengineering and Morphogenesis

CANADA

Alberta
EDMONTON

CANADA

Ontario
OTTAWA

Finanziato da un ente associato a IRDiRCGenetic Regulation of Myogenesis
Ottawa Hospital Research Institute
Sprott Center for Stem Cell Research

CANADA

Ontario
TORONTO

CANADA

Québec
ADDRESS: NOT PROVIDED - CA

CANADA

Québec
ADDRESS: NOT PROVIDED - CA

Finanziato da un ente associato a IRDiRCElucidation of Wnt7a mechanism of action for muscle regeneration
Institution: Information not provided - CA

CANADA

Québec
ADDRESS: NOT PROVIDED - CA

CANADA

Québec
ADDRESS: NOT PROVIDED - CA

CANADA

Québec
ADDRESS: NOT PROVIDED - CA

CANADA

Québec
JONQUIÈRE

Finanziato da un ente associato a IRDiRCMotor, 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

Finanziato da un ente associato a IRDiRCStrategies for therapy of respiratory muscle failure in muscular dystrophy
Meakins-Christie Laboratories
Meakins-Christie Labs

CANADA

Québec
QUÉBEC

Finanziato da un ente associato a IRDiRCCorrection 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

Finanziato da un ente associato a IRDiRCCan 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

CIPRO

Cyprus
ADDRESS: NOT PROVIDED - CY

Finanziato da un ente associato a IRDiRCLNA/2'OMe mixmers against toxic CUG expanded RNA
Institution: Information not provided - CY

FRANCIA

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCIA

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCIA

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

Finanziato da un ente associato a IRDiRCTranslational suppression of nonsense mutations found in DMD
Institution: Information not provided - FR

FRANCIA

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

Finanziato da un ente associato a IRDiRCVenous thromboembolism in myotonic dystrophy type 1
Institution: Information not provided - FR

FRANCIA

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCIA

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCIA

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCIA

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCIA

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCIA

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCIA

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCIA

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCIA

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCIA

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCIA

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCIA

AUVERGNE-RHONE-ALPES
BRON

FRANCIA

AUVERGNE-RHONE-ALPES
PRAGUE

Finanziato da un ente associato a IRDiRCPreclinical 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

FRANCIA

ILE-DE-FRANCE
EVRY

FRANCIA

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

FRANCIA

ILE-DE-FRANCE
EVRY

FRANCIA

ILE-DE-FRANCE
GIF-SUR-YVETTE

Finanziato da un ente associato a IRDiRCRescue_ribosome : Rescue Pathways for unrecycled ribosomes - FR
Institut de Biologie Intégrative de la Cellule (I2BC)
Département Biologie des Génomes

FRANCIA

ILE-DE-FRANCE
GIF-SUR-YVETTE

Finanziato da un ente associato a IRDiRCRestoration of Ca2+-signalling in mdx mice by targeting the endo-lysosmal two-pore channel (TPC)
Institut des Neuroscieces Paris-Saclay
Code neuronal & perception auditive

FRANCIA

ILE-DE-FRANCE
PARIS

Implementation of non-invasive prenatal diagnosis of rare and severe monogenic diseases
CHU Paris Centre - Hôpital Cochin, Site Cochin
Service de génétique et biologie moléculaires

FRANCIA

ILE-DE-FRANCE
PARIS

Finanziato da un ente associato a IRDiRCDM1 disease mechanisms in the central nervous system: from brain cell-specific pathogenesis to misregulated glutamate homeostasis
Centre de Psychiatrie et Neurosciences
Neurobiologie du vieillissement normal et pathologique

FRANCIA

ILE-DE-FRANCE
PARIS

Finanziato da un ente associato a IRDiRCGene therapy in DM1 cells by induction of a TALE Nuclease
Hôpital Necker-Enfants Malades
Service de Génétique Moléculaire

FRANCIA

ILE-DE-FRANCE
PARIS

Finanziato da un ente associato a IRDiRCDM_Neuroglia : RNA toxicity and neuroglial miscommunication in myotonic dystrophy brains - FR
IMAGINE - Institut des Maladies Génétiques
Equipe "Triplets CTG instables et dystrophie myotonique"

FRANCIA

ILE-DE-FRANCE
PARIS

Finanziato da un ente associato a IRDiRCHow to contract CAG.CTG repeats in myotonic dystrophy type 1
IMAGINE - Institut des Maladies Génétiques
Laboratoire "Triplets CTG instables et dystrophie myotonique"

FRANCIA

ILE-DE-FRANCE
PARIS

FRANCIA

ILE-DE-FRANCE
PARIS

Finanziato da un ente associato a IRDiRCiDM-scope: the international French-Quebec myotonic dystrophy registry
Institut de Myologie - Hôpital Pitié-Salpêtrière
Unité clinique de pathologie neuromusculaire

FRANCIA

ILE-DE-FRANCE
VERSAILLES

Finanziato da un ente associato a IRDiRCElucidate 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

FRANCIA

OCCITANIE
MONTPELLIER

FRANCIA

OCCITANIE
MONTPELLIER

Finanziato da un ente associato a IRDiRCTowards 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

FRANCIA

OCCITANIE
MONTPELLIER

Finanziato da un ente associato a IRDiRCCardiac 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

FRANCIA

PAYS DE LA LOIRE
NANTES

Finanziato da un ente associato a IRDiRCTOLGEN: In vivo AAV-based gene transfer to the muscle: Deciphering host immune responses - FR
IRS2 - Nantes Biotech
Thérapie Génique Translationnelle des Maladies Génétiques

GERMANIA

Berlin
BERLIN

Finanziato da un ente associato a IRDiRCTAMDMD: Tamoxifen in Duchenne muscular dystrophy - a randomised placebo controlled phase 2 trial - DE
DRK Kliniken Berlin Westend
Klinik für Kinder- und Jugendmedizin

GIAPPONE

JAPAN
NAGANO

Finanziato da un ente associato a IRDiRCResearch on natural history of Becker muscular dystrophy for establishment of preventive medicine
National Hospital Organization Matsumoto Medical Center
Department of Neurology

GIAPPONE

JAPAN
OKAYAMA

Finanziato da un ente associato a IRDiRCResearch for practical application of an innovative peptide drug for DMD
Kawasaki Medical School
Department of Neurology

GIAPPONE

JAPAN
OSAKA

Finanziato da un ente associato a IRDiRCEvidence-generating clinical research for myotonic dystrophy
Osaka University Graduate School of Medicine
Department of Functional Diagnostic Science

GIAPPONE

JAPAN
TOKYO

Finanziato da un ente associato a IRDiRCDevelopment of a novel peptide-conjugated phosphorodiamidate morpholino therapy for Duchenne muscular dystrophy.
National Center of Neurology and Psychiatry
Department of Molecular Therapy

ITALIA

EMILIA ROMAGNA
ADDRESS: NOT PROVIDED - IT

ITALIA

EMILIA ROMAGNA
ADDRESS: NOT PROVIDED - IT

ITALIA

LAZIO
ROMA

Finanziato da un ente associato a IRDiRCRole of Dystrophin-associated protein complex (DPC) in intracellular signaling and trafficking pathways
ISS - Istituto Superiore di Sanità
Centro Nazionale Malattie Rare

ITALIA

LAZIO
ROMA

Finanziato da un ente associato a IRDiRCAnalysis 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

ITALIA

LOMBARDIA
MILANO

Finanziato da un ente associato a IRDiRCForme ossidata e ridotta di HGMB1: nuovi bersagli terapeutici nella distrofia muscolare di Duchenne
IRCCS Ospedale San Raffaele
Unit of Chromatin Dynamics - division of Genetics and Cell Biology

ITALIA

VENETO
PADOVA

Finanziato da un ente associato a IRDiRCValutazione pre-clinica di nanoparticelle biocompatibili come sistema di trasporto di oligoribonucleotidi antisenso per indurre il ripristino di distrofina tramite "exon skipping"
Università degli Studi di Padova- Polo A.Vallisneri
Dipartimento di Istologia, Microbiologia e Biotecnologie Biomediche

ITALIA

VENETO
PADOVA

Finanziato da un ente associato a IRDiRCIl ruolo di Raptor/mTORC1 nel muscolo scheletrico adulto
Venetian Institute of Molecular Medicine
Dipartimento di Scienze Biomediche

PAESI BASSI

Gelderland
NIJMEGEN

PAESI BASSI

Gelderland
NIJMEGEN

PAESI BASSI

Zuid-Holland
LEIDEN

PORTOGALLO

CENTRO
ADDRESS: NOT PROVIDED - PT

REGNO UNITO; GRAN BRETAGNA

Edinburgh
EDINBURGH

REGNO UNITO; GRAN BRETAGNA

Oxfordshire
OXFORD

Finanziato da un ente associato a IRDiRCMICA: The role of utrophin in DMD and its therapeutic potential
University of Oxford
Department of Physiology, Anatomy and Genetics

REGNO UNITO; GRAN BRETAGNA

Tyne & Wear
NEWCASTLE UPON TYNE

TREAT-NMD: Accelerating Treatments for Neuromuscular Diseases (coordination)
Newcastle upon Tyne Hospitals NHS Trust
John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine

SPAGNA

Cataluña
BADALONA

Finanziato da un ente associato a IRDiRCDIMINUTES, Childhood and adult myotonic dystrophy: evaluation of new treatments and pathogenicity through genetic, epigenetic and molecular imaging analysis
Fundació Institut d'Investigació en Ciències de la Salut Germans Trias i Pujol
Unidad de Investigación Neuromuscular y Neuropediátrica

SPAGNA

Cataluña
BARCELONA

Characterization of the role of fibroadipogenic precursors in the degenerative process of muscular dystrophies
Hospital de la Santa Creu i Sant Pau
Unidad funcional de enfermedades autoinmunitarias sistémicas

SPAGNA

Comunidad Valenciana
VALENCIA

Finanziato da un ente associato a IRDiRCTherapeutic modulation of MBNL genes as innovative treatments for myotonic dystrophy
INCLIVA - Facultad de Medicina de la Universidad de Valencia
Grupo de investigación en genómica traslacional

SPAGNA

Comunidad Valenciana
VALENCIA

Finanziato da un ente associato a IRDiRCPreclinical development of an innovative drug for myotonic dystrophy
INCLIVA - Facultad de Medicina de la Universidad de Valencia
Grupo de investigación en genómica traslacional

SPAGNA

Madrid
MADRID

SPAGNA

País Vasco
SAN SEBASTIÁN

Finanziato da un ente associato a IRDiRCTherapeutic potential of Ahulken compounds against cognitive and muscle deficits in Duchenne muscular dystrophy
Instituto de Investigación Sanitaria Biodonostia
Área de Neurociencias

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

Finanziato da un ente associato a IRDiRCSTAT3 signaling network in MuSCs as therapeutic target for DMD
Institution: Information not provided - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

Finanziato da un ente associato a IRDiRCWhole body single aav microgene therapy in canine dmd
Institution: Information not provided - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

Finanziato da un ente associato a IRDiRCTherapeutics for rare and neglected diseases - science
Institution: Information not provided - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

Finanziato da un ente associato a IRDiRCGene therapy platform for rare diseases
Institution: Information not provided - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

Finanziato da un ente associato a IRDiRCMicrotubule regulated mechanotransduction in skeletal muscle
Institution: Information not provided - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

Finanziato da un ente associato a IRDiRCMuscle tregs in health and disease
Institution: Information not provided - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

Finanziato da un ente associato a IRDiRCRna toxicity and muscle regeneration
Institution: Information not provided - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

Finanziato da un ente associato a IRDiRCSarcolipin in duchenne muscular dystrophy
Institution: Information not provided - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

Finanziato da un ente associato a IRDiRCBioinformatics and genomics
Institution: Information not provided - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

Finanziato da un ente associato a IRDiRCHighthroughput screening and cell repository
Institution: Information not provided - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

Finanziato da un ente associato a IRDiRCOptimization of ao drugs 45; 51 & 53
Institution: Information not provided - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

Finanziato da un ente associato a IRDiRCBiomarker discovery for ao accumulation in kidney
Institution: Information not provided - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

Finanziato da un ente associato a IRDiRCClinical evaluation of urine biomarkers for morpholino
Institution: Information not provided - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

Finanziato da un ente associato a IRDiRCPreclinical dosing optimization: dosing schedule; tissue
Institution: Information not provided - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

Finanziato da un ente associato a IRDiRCStrategies to overcome immunity in gene therapy of dmd
Institution: Information not provided - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

CANADA

Ontario
TORONTO

FRANCIA

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

Finanziato da un ente associato a IRDiRCRole of annexins in membrane repair of human skeletal muscle
Institution: Information not provided - FR

FRANCIA

AUVERGNE-RHONE-ALPES
CLERMONT-FERRAND

FRANCIA

ILE-DE-FRANCE
EVRY

FRANCIA

ILE-DE-FRANCE
PARIS

Finanziato da un ente associato a IRDiRCRole 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"

FRANCIA

ILE-DE-FRANCE
PARIS

Finanziato da un ente associato a IRDiRCSTaHR : Stimulation of Targeted Homologous Recombination for Gene Therapy - FR
Muséum National d'Histoire Naturelle
Laboratoire Structure et Instabilité des Génomes - INSERM U1154 - CNRS 7196

FRANCIA

PAYS DE LA LOIRE
NANTES

Finanziato da un ente associato a IRDiRCFROGH : FRench Regional Origins in Genetics for Health - FR
Institut de Recherche en Santé - Université de Nantes
L'Institut du thorax - Inserm UMR 1087 / CNRS UMR 6291

GIAPPONE

JAPAN
HYOGO

Finanziato da un ente associato a IRDiRCDevelopment ofa new molecular targeted therapy for Fukuyama muscular dystrophy
Kobe University Graduate School of Medicine
Department of Pediatrics

GIAPPONE

JAPAN
TOKYO

Finanziato da un ente associato a IRDiRCFormation of integrated omics analysis base of refractory diseases in children and perinatal period
National Center for Child Health and Development
Research Institute

ITALIA

LAZIO
ROMA

Finanziato da un ente associato a IRDiRCMalattie rare causate dalla deregolazione funzionale delle proteine Rho GTPasi
ISS - Istituto Superiore di Sanità
Centro Nazionale Malattie Rare

ITALIA

LIGURIA
GENOVA

Finanziato da un ente associato a IRDiRCATP extracellulare e cellule T regolatorie: nuovi bersagli terapeutici nella Distrofia Muscolare dei Cingoli da deficit di alfa-Sarcoglicano (LGMD2D)
IRCCS Istituto G. Gaslini - Ospedale Pediatrico
U.O.C. Neurologia pediatrica e Malattie muscolari

NORVEGIA

Nord-Norge
TROMSØ

Pathophysiological mechanisms with Limb Girdle muscular dystrophy Type 21
UNN - Universitetssykehuset Nord-Norge
NMK - Nevromuskulært kompetansesenter

NORVEGIA

Nord-Norge
TROMSØ

Limb-Girdle Muscular Dystrophy in Norway, a cohort study
UNN - Universitetssykehuset Nord-Norge
NMK - Nevromuskulært kompetansesenter

REGNO UNITO; GRAN BRETAGNA

Tyne & Wear
NEWCASTLE-UPON-TYNE

Defining Clinical Endpoints in Limb Girdle Muscular Dystrophy (LGMD) (GRASP)
Newcastle University
Newcastle Clinical Trials Unit

SPAGNA

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

SPAGNA

País Vasco
SAN SEBASTIÁN

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

Finanziato da un ente associato a IRDiRCA powerful web-based discovery platform for rare disease genetics
Institution: Information not provided - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

Finanziato da un ente associato a IRDiRCThe sarcoglycan complex in skeletal muscle mechanotransduction
Institution: Information not provided - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

Finanziato da un ente associato a IRDiRCThe role of dystroglycan in neural circuit development.
Institution: Information not provided - US

STATI UNITI

Washington
ADDRESS: NOT PROVIDED - US

Progetti di ricerca multicentrici