Orphanet: Search by disease/gene
x

Search for a research project

* (*) mandatory field

237 Result(s)

Funded by an IRDiRC member =

Research projects

FRANCE

ILE-DE-FRANCE
EVRY

Cartography and genetic identification of genes involved in child motor neuropathies
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

Understanding the molecular mechanisms underlying familial dysautonomia
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberPre-clinical development of an AAV-mediated gene therapy for type-II SMA
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberStem cell Therapy for Retinal Epithelium replacement Assay in Monogenic retinopathies
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Toward gene repair for Huntington's disease
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberOxidative stress: the main pathogenic process in myositis?
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberGenetics of Young Amyotrophic Lateral Sclerosis
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberRole of the Ca2+ activated Cl- channel, Anoctamin 6, in motoneuron pathophysiology
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberTargeting Schwann cell proteostasis as a therapeutic strategy in Charcot-Marie-Tooth disease
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberDissecting the retrograde signalling that controls neuromuscular Junction
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberRole of Calcium Handling Proteins and Calcineurin Signaling in Myotonic Dystrophy type 1
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberPrion protein dysfunction in inherited metabolic myopathies
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberControl and modulation of myogenic differentiation of stem cells by using optogenetic approaches
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberGene transfer tolerance in combined liver and muscle rAAV gene therapy
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberTranslational suppression of nonsense mutations found in DMD
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberVenous thromboembolism in myotonic dystrophy type 1
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberAspartylglucosaminuria gene therapy using AAV vectors to target the CNS
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberA gene therapy approach for treating CMT4C
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberNeurofilaments in Health and Charcot-Marie-Tooth diseases
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberRole of Actin-SRF-MRTFA Axis in the development of dilated cardiomyopathy
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberElucidation of Wnt7a mechanism of action for muscle regeneration
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberMolecular mediators of the spinal muscular atrophy NMJ phenotype
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberAbnormal mitoribosomal biogenesis and protein maturation in human mitochondrial diseases.
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberHuman reserve cells: a dynamic tool to discover genes involved in muscle stem cell quiescence
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberPathomechanisms in Andersen's syndrome : insights into excitable and non excitable tissues
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberUnderstanding the developmental onset of muscular dystrophy in a mouse model of MDC1A.
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberEstablishing new models for primary dystroglycanopathies
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberDystrophin-deficient diaphragm dysfunction induced by mechanical ventilation in mdx mice.
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberPathophysiology of congenital muscle weakness linked to abnormal neuromuscular excitability
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberThe role of axonal metabolic changes in the pathophysiology of Charcot-Marie-Tooth disease
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberModelling FSHD as a tool for testing therapeutics
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberUnraveling the mode of action of Mitofusins in healthy and pathogenic mitochondrial fusion
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberModeling infantile neuroaxonal dystrophy, a nalcn channel-related disorder in zebrafish
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberInduced pluripotent stem cells to treat pelizaeus merzbacher disease
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberInfluence of mtDNA background (haplogroups) on mitochondrial pathologies.
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberSIX homeoproteins and muscle stem cells properties
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberRole of annexins in membrane repair of human skeletal muscle
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberRegulation and morphogenesis of cranial muscle derivatives
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberMisglycosylation in Charcot-Marie-Tooth neuropathies associated to MPZ mutations
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberNeonatal stem cell gene therapy for severe Mucopolysaccharidosis type II
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberRigorous, integrated miRNA-DNA plasma biomarkers for amyotrophic lateral sclerosis
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberUncovering the function of HDAC4 in skeletal muscle
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberGene therapy for Wolfram Syndrome
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberUnravelling mechanisms of axonal loss in late-onset genetic neuropathies
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberTargeting of Histone Deacetylase 6 (HDAC6) in Neuromuscular Diseases
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberUnravelling POIKTMP pathophysiology for design of therapeutic approaches
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberNeuroprotective role of CYP46A1 in Huntington’s disease using gene transfer therapy in brain
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberTargets for heart regeneration : mechanisms controlling cardiomyocyte proliferation
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberLiver Gene Transfer-Induced allogeneic Tolerance for organ transplantation and cell therapy
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberEffect of Rituximab therapy on T-cell repertoire in MuSK positive Myasthenia Gravis
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberUpper Limbs evaluation in hereditary NeuropAthies: the ULNA project
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberStrategies to enhance the efficacy of enzyme replacement and gene therapy in Pompe disease
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberZebrafish as a model system for GNE Myopathy
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberASC-1, a novel actor in the pathophysiology of congenital muscle disorders
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberMitochondrial myopathies with mtDNA instability: the role of cristae maintenance
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberLNA/2'OMe mixmers against toxic CUG expanded RNA
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberRole of PTX3 in the pathogenesis of idiopathic inflammatory myopathies
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberEpigenetic modulation of pro-inflammatory cell stress in inclusion body myositis
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberAnalysis of the DM2 pathogenic mechanisms using Drosophila as model system
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberPin1: a new potential target to induce slow fiber conversion in Duchenne Muscular Dystrophy
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberRepurposing drugs that target eEF1A2 to increase translation of utrophin in dystrophic muscle
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberMechanosensitive ionic channels in zebrafish heart regeneration
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberSELNAC: a first therapeutic trial for SEPN1-related myopathy
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberRole of Tenascin-C in muscle stem cell function
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberSTAT3 signaling network in MuSCs as therapeutic target for DMD
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberControl of satellite cell self-renewal by Sonic hedgehog signalling and primary cilia.
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberAcetylcholine receptor binding B cells as biomarker for myasthenia gravis
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberRNA processing role in muscle degeneration opens therapeutical options for adult myopathies
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberPreclinical assessment of an antigen-specific therapeutic approach for MG
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberCharacterization of Liver-induced allo-specific CD8 regulatory T lymphocytes
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberBone morphogenetic proteins regulate patterning of limb muscles.
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberMolecular mechanisms of chronic muscle pain - Focus on Acid-Sensing Ion Channels (ASICs)
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberWhite adipose tissue: a stem/stromal cell provider involved in muscle regeneration?
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberDystrophin-deficient diaphragm dysfunction induced by mechanical ventilation in mdx mice.
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberRole of TRF2, a key telomere complex, in skeletal muscle homeostasis
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberRole of LRRFIP2 in the control of heart development and skeletal muscle regeneration
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberA new mouse model for desminopathies: physiopathological mechanisms, muscle repair and therapy
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberFAT1 Signaling in skeletal muscle growth and repair: Muscle versus mesenchyme perspectives
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberStudy of the multiple functions of Nfix in Muscular Dystrophies: a focus on macrophage biology
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberUse of a novel in vitro DMD model to study muscle fusion during health and disease
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberNEXTGEN-AAV: Development of next-generation AAV vectors for Duchenne muscular dystrophy
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberFunction of LIX1 and its interacting partner during digestive smooth muscle plasticity
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberHMGB1 Redox Forms as New Targets in Duchenne Muscular Dystrophy
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberRNA-based therapeutics for OTC deficiency
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberThe role of Raptor/mTORC1 in adult skeletal muscle
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberUpper Limbs evaluation in hereditary NeuropAthies: the ULNA project (Partner 2)
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberStabilization of tRNAs as a therapeutic strategy for diseases due to mutations in mt-tRNAs
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberUpper Limbs evaluation in hereditary NeuropAthies: the ULNA project (Partner 3)
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberUpper Limbs evaluation in hereditary NeuropAthies: the ULNA project (Partner 4)
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberUpper Limbs evaluation in hereditary NeuropAthies: the ULNA project (Partner 5)
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberUpper Limbs evaluation in hereditary NeuropAthies: the ULNA project (coordination)
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberFrench National Registry for FacioScapuloHumeral muscular Dystrophy (FSHD)
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberResolve FSHD: clinical trial readiness to solve barriers to drug development in FSHD
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberiDM-scope: the international French-Quebec myotonic dystrophy registry
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberBcl11b/CTIP2, a transcriptional repressor: its role in cardiac homeostasis and remodeling
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberRole of the calcium-activated chloride channel TMEM16F in amyotrophic lateral sclerosis
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberMitochondrial gene expression in normal, mutant and 3-parent preimplantation embryos
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberCell signaling, Reinnervation and Metabolism in Amyotrophic Lateral Sclerosis (CERMALS)
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberConsolidating data for rescue of missense mutations from degradation
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberPathophysiology and therapeutic proof-of-concepts for congenital myopathies
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberA CRISPR-C2c2 based system to target toxic RNA in myotonic dystrophy type 1
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberCharacterization of the phenotypic variability in FSHD families for assisting clinical research
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberIntermuscular adipose tissue as a trigger of muscle wasting?
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberThe bacterial protein CNF1 as a novel strategy to counteract mitochondrial encephalomyopathies
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberThe effect of autophagy induction on the neuromuscular junction in ALS
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberAddressing Complex I deficiencies through drug screening
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberDefining new functions for the SMN complex from yeast to human
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberDeveloping combinatorial therapies for the treatment of spinal muscular atrophy
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberGene therapy in DM1 cells by induction of a TALE Nuclease
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberFunctional analysis of separase-dependent lamins' regulation in AD-EDMD
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberGenetic and epigenetic control of adult muscle fiber phenotype
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberHow to contract CAG.CTG repeats in myotonic dystrophy type 1
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberIgG4 in inflammatory neuropathies: pathogenic effects and novel targets
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberInvestigating heterogeneities and morphogenesis of cranial mesoderm derived skeletal muscles
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberMolecular mechanisms of mtDNA maintenance in human health and disease
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberMuSK intracellular pathways in congenital myasthenic syndromes
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberPhysiopathology of muscle atrophy in myotonic dystrophy
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberRestoration of Ca2+-signalling in mdx mice by targeting the endo-lysosmal two-pore channel (TPC)
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberUnravelling mechanisms of axonal loss in late-onset genetic neuropathies - partner 1
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberStructural studies of cardiac myosin for therapeutical approaches against HCM
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberwhole body periodic acceleration a novel treatment for duchenne cardiomyopathy in mdx mice
AFM Téléthon - Association Française contre les Myopathies

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberTarget specific antibody-siRNA conjugate therapy for experimental myasthenia
AFM Téléthon - Association Française contre les Myopathies