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Research projects

CANADA

Ontario
HAMILTON

Funded by an IRDiRC memberRole of Xin, an actin-binding protein, in satellite cells and muscular dystrophies
McMaster University
Department of Pathology and Molecular Medicine

FRANCE

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCE

ILE-DE-FRANCE
VERSAILLES

Funded by an IRDiRC memberThe 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

Funded by an IRDiRC memberUnravelling 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

ITALY

LAZIO
ROMA

Funded by an IRDiRC memberRole of miR-200c in dystrophic muscle regeneration of mdx mice and DMD patients
Istituto Dermopatico dell'Immacolata - IRCCS
I Divisione Dermatologia

ITALY

LAZIO
ROMA

Funded by an IRDiRC memberIn 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

Funded by an IRDiRC memberInterventions in genetic counseling
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberRegulation of subcellular organization in skeletal muscle
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

AUSTRIA

WIEN
WIEN

Funded by an IRDiRC memberLong-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

CANADA

Alberta
EDMONTON

Funded by an IRDiRC memberTargeting RNA Splicing for the Treatment of Duchenne Muscular Dystrophy
University of Alberta
Department of Medical Genetics

CANADA

Ontario
OTTAWA

Funded by an IRDiRC memberDefining 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

Funded by an IRDiRC memberGenetic Regulation of Myogenesis
Ottawa Hospital Research Institute
Sprott Center for Stem Cell Research

CANADA

Ontario
OTTAWA

Funded by an IRDiRC memberStimulating cilia-mediated Hedgehog signaling to restore dystrophin-deficient satellite cell function
Ottawa Hospital Research Institute
Sprott Center for Stem Cell Research

CANADA

Ontario
TORONTO

CANADA

Québec
ADDRESS: NOT PROVIDED - CA

Funded by an IRDiRC memberElucidation 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
MONTRÉAL

Funded by an IRDiRC memberUsing 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

Funded by an IRDiRC memberContribution 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

Funded by an IRDiRC memberStrategies for therapy of respiratory muscle failure in muscular dystrophy
Meakins-Christie Laboratories
Meakins-Christie Labs

CANADA

Québec
QUÉBEC

Funded by an IRDiRC memberCorrection 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

Funded by an IRDiRC memberCan 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

Funded by an IRDiRC memberMultifunctional properties of osteoprotegerin in muscular dystrophy and muscle repair
CHUQ - (CHUL) Centre hospitalier de l'Université Laval
Axe Neuosciences, CHUL

FRANCE

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCE

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCE

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCE

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

Funded by an IRDiRC memberSIX homeoproteins and muscle stem cells properties
Institution: Information not provided - FR

FRANCE

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCE

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCE

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCE

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCE

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

Funded by an IRDiRC memberDuchenne Muscular Dystrophy non-viral gene therapy - FR
Institution: Information not provided - FR

FRANCE

ILE-DE-FRANCE
CORBEIL-ESSONNES

Funded by an IRDiRC memberGMP production for rare diseases
YposKesi (AFM-Telethon-Genethon)

FRANCE

ILE-DE-FRANCE
GIF-SUR-YVETTE

Funded by an IRDiRC memberRescue_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

Funded by an IRDiRC memberRestoration 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

Funded by an IRDiRC memberMYOCITY - A multidimensional single-cell approach to understand muscle dystrophy - FR
CLCC Institut Curie
Labex Dendritic Cell Biology (DCBIOL)

FRANCE

ILE-DE-FRANCE
PARIS

Funded by an IRDiRC memberDecoding 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

Funded by an IRDiRC memberNovel 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

Funded by an IRDiRC memberSpatio-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

Funded by an IRDiRC memberInduction 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

Funded by an IRDiRC memberElucidate 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

Funded by an IRDiRC memberElucidate 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

Funded by an IRDiRC memberFunctional 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

Funded by an IRDiRC memberTowards 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

Funded by an IRDiRC memberCardiac 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

Funded by an IRDiRC memberExploring 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

Funded by an IRDiRC memberRole 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

Berlin
BERLIN

Funded by an IRDiRC memberTAMDMD: 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

ITALY

EMILIA ROMAGNA
ADDRESS: NOT PROVIDED - IT

ITALY

LAZIO
ROMA

Funded by an IRDiRC memberExosome-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

Funded by an IRDiRC memberNew 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

Funded by an IRDiRC memberFunctionalized nanoparticles for targeted genome editing in Duchenne Muscular Dystrophy - IT
IRCCS Fondazione Santa Lucia
Laboratorio di Epigenetica e Trasduzione del Segnale

ITALY

LAZIO
ROMA

Funded by an IRDiRC memberRole of Dystrophin-associated protein complex (DPC) in intracellular signaling and trafficking pathways
ISS - Istituto Superiore di Sanità
Centro Nazionale Malattie Rare

ITALY

LAZIO
ROMA

Funded by an IRDiRC memberRole of glia in neurological comorbidities of Duchenne muscular dystrophy - IT
Sapienza Università di Roma
FISIOLOGIA E FARMACOLOGIA "VITTORIO ERSPAMER"

ITALY

LOMBARDIA
MILANO

Funded by an IRDiRC memberNovel satellite cell heterogeneity in healthy and pathological regeneration - IT
Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico
Laboratorio Cellule Staminali

ITALY

LOMBARDIA
MILANO

Funded by an IRDiRC memberHMGB1 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

Funded by an IRDiRC memberMYOCITY - 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

Funded by an IRDiRC memberAn 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

Funded by an IRDiRC memberTowards 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)

Funded by an IRDiRC memberTowards 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

ITALY

VENETO
PADOVA

Funded by an IRDiRC memberRole of Cyclophilins in Duchenne Muscular Dystrophy
Università degli Studi di Padova- Polo A.Vallisneri
Dipartimento di Scienze Biomediche

ITALY

VENETO
PADOVA

Funded by an IRDiRC memberTargeting the Mitochondrial Calcium Uniporter to counteract Duchenne Muscular Dystrophy
Università degli Studi di Padova- Polo A.Vallisneri
Dipartimento di Scienze Biomediche

NETHERLANDS

Gelderland
NIJMEGEN

NETHERLANDS

Gelderland
NIJMEGEN

SPAIN

Cataluña
BADALONA

Funded by an IRDiRC memberEXPLORING 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

Funded by an IRDiRC memberNouvelles stratégies pour améliorer la dystrophie musculaire de Duchenne
Universitat Pompeu Fabra. Campus del Mar
Grupo de Biología Celular

SPAIN

Madrid
MADRID

Funded by an IRDiRC memberFunctionalized nanoparticles for targeted genome editing in Duchenne Muscular Dystrophy - ES
IMDEA Nano: Madrid Institute of Advanced Studies in Nanoscience

SWITZERLAND

Suisse Alémanique
BASEL

SWITZERLAND

Suisse Romande
GENÈVE

UNITED KINGDOM

Northamptonshire
NORTHAMPTON

Funded by an IRDiRC memberRNA 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

Massachusetts
BOSTON

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberSTAT3 signaling network in MuSCs as therapeutic target for DMD
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberWhole body single aav microgene therapy in canine dmd
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberTherapeutics for rare and neglected diseases - science
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberGene therapy platform for rare diseases
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberMicrotubule regulated mechanotransduction in skeletal muscle
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberMuscle tregs in health and disease
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberSarcolipin in duchenne muscular dystrophy
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberBioinformatics and genomics
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberHighthroughput screening and cell repository
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberOptimization of ao drugs 45; 51 & 53
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberBiomarker discovery for ao accumulation in kidney
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberClinical evaluation of urine biomarkers for morpholino
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberPreclinical dosing optimization: dosing schedule; tissue
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberStrategies to overcome immunity in gene therapy of dmd
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

CANADA

Alberta
CALGARY

Funded by an IRDiRC memberIdentifying Cellular and Molecular Modifiers of Muscular Dystrophy
University of Calgary
Department of Biochemistry and Molecular Biology

CANADA

Ontario
OTTAWA

Funded by an IRDiRC memberDifferential role of myogenic regulatory factors in establishing muscle-specific gene expression
Ottawa Hospital Research Institute
Sprott Center for Stem Cell Research

CANADA

Ontario
OTTAWA

Funded by an IRDiRC memberSatellite 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

Funded by an IRDiRC memberSIX family transcription factors in adult muscle regeneration.
University of Ottawa
Faculty of Medicine -

FRANCE

ILE-DE-FRANCE
PARIS

FRANCE

OCCITANIE
TOULOUSE

Funded by an IRDiRC memberIntermuscular adipose tissue as a trigger of muscle wasting?
Institut des Maladies Métaboliques et Cardiovasculaires
Laboratoire de recherche sur les obésités

ISRAEL

ISRAEL
REHOVOT

Funded by an IRDiRC memberMechanical 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

ITALY

LAZIO
ROMA

ITALY

LOMBARDIA
MILANO

ITALY

PIEMONTE
TORINO

Funded by an IRDiRC memberGLUD1 as a potential target in Muscular Dystrophy
Università degli Studi di Torino
Dipartimento di Biotecnologie Molecolari e Scienze Della Vita

JAPAN

JAPAN
OSAKA

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

Funded by an IRDiRC memberIDOLS-G: Improved diagnostic output in large sarcomeric genes - EE
Department of Clinical Genetics - University of Tartu
Department of Clinical Genetics

FINLAND

Finland
HELSINKI

FRANCE

GRAND-EST
ILLKIRCH-GRAFFENSTADEN

Funded by an IRDiRC memberIDOLS-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

Funded by an IRDiRC memberIDOLS-G: Improved diagnostic output in large sarcomeric genes - HU
Semmelweis University
Department of Biophysics and Radiation Biology

ITALY

TOSCANA
SESTO FIORENTINO (FIRENZE)

Funded by an IRDiRC memberIDOLS-G: Improved diagnostic output in large sarcomeric genes - IT
Università degli Studi di Firenze
Dipartimento di Biologia

NETHERLANDS

Noord-Holland
AMSTERDAM

Funded by an IRDiRC memberIDOLS-G: Improved diagnostic output in large sarcomeric genes - NL
Amsterdam UMC, locatie VUmc
Afdeling Fysiologie

SPAIN

Cataluña
BARCELONA

Funded by an IRDiRC memberIDOLS-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

BELGIUM

ANTWERPEN
ANTWERPEN

Funded by an IRDiRC memberA 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

FRANCE

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCE

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCE

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCE

AUVERGNE-RHONE-ALPES
GRENOBLE

Funded by an IRDiRC memberEarly 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

Funded by an IRDiRC memberInterplay 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

Funded by an IRDiRC memberAmino acid metabolism and alternative splicing
ENS - Ecole normale supérieure de Lyon
Laboratoire de biologie et modélisation de la cellule - UMR5239

FRANCE

ILE-DE-FRANCE
CRÉTEIL

FRANCE

ILE-DE-FRANCE
CRÉTEIL

Funded by an IRDiRC memberAn integrated translational program for neuromuscular disorders
Hôpitaux Universitaires Henri Mondor
Centre expert de maladie neuro-musculaire

FRANCE

ILE-DE-FRANCE
GARCHES

Funded by an IRDiRC memberPatient-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
PARIS

FRANCE

ILE-DE-FRANCE
PARIS

Funded by an IRDiRC membersPAM: 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

FRANCE

PROVENCE-ALPES-COTE D'AZUR
MARSEILLE

Funded by an IRDiRC memberModelisation 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

Funded by an IRDiRC memberA 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

ITALY

LAZIO
ROMA

Funded by an IRDiRC memberCellular network driving neuromuscular junction stability
Sapienza Università di Roma
Università degli Studi di Roma "La Sapienza"

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
ISGlobal - Instituto de Salud Global de Barcelona
Programa de investigación en Enfermedades no transmisibles y medio ambiente

SPAIN

Madrid
MADRID

UNITED STATES

California
PALO ALTO

Funded by an IRDiRC membersPAM: Developing peptide-based drugs to target ataxin-2 in neuromuscular disease - US
Stanford University School of Medicine
Department of Genetics

CANADA

Colombie-Britannique
VICTORIA

CANADA

Ontario
TORONTO

ITALY

LAZIO
ROMA

Funded by an IRDiRC memberDiverse rare diseases caused by functional dysregulation of Rho GTPase-related proteins
ISS - Istituto Superiore di Sanità
Centro Nazionale Malattie Rare

SPAIN

Madrid
MADRID

Funded by an IRDiRC memberIdentification 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 STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberA powerful web-based discovery platform for rare disease genetics
Institution: Information not provided - US

Multicentric Research projects