Gene therapy Regeneration Next generation sequencing Heart failure Laminopathies Muscle CSF protein Rare neuromuscular diseases CMTX Cancer Ehlers‐Danlos Syndrome Allele‐specific silencing therapy Hypermobile EDS Cancer biomarkers Dynamin 2 Emery-Dreifuss muscular dystrophy Therapy COL6A3 Collagen VI-related myopathies NGS collagen type VI congenital muscular dystrophy CMD limb-girdle muscular dystrophy LGMD muscular MRI neuromuscular disorders Myogenesis Nuclear envelope COVID-19 A-type lamin Actionability GNE LGMD POPDC1 Cardiomyopathy Cardiac conduction system Angiotensin-converting enzyme inhibitor Allele-specific silencing therapy BiP Myotubes Maladies rares et orphelines Treatment Centronuclear myopathy Base de données FAIR AAV CRISPR Calcium handling Myopathies Angiotensin-converting enzyme inhibitors Lamin A/C LMNA gene Clinical trial A-type lamins Muscular dystrophy Myologie Rare diseases Heart LMNA gene Diagnosis Treatment delay Laminopathie Autophagosome maturation RNA interference Laminopathy Actionable gene IPSC C elegans LMNA Mouse INPP5K Exome Neuromuscular diseases Dilated cardiomyopathy Acetyltransferase Butyrylcholinesterase Lamin A/C nuclei Becker muscular dystrophy BMD Duchenne muscular dystrophy DMD miRNA nNOS Muscle MRI Connective tissue Muscle biopsy Dystrophie musculaire Maladies rares Duchenne muscular dystrophy C2C12 Myopathy Errance diagnostique COL6A1 BVES Lamins Titin Alternative splicing Becker muscular dystrophy Emerin Dystrophine LMNA-related congenital muscular dystrophy Patient registry Cardiology Lamin A/C Mutations Biomarker Adult SMA Allele-specific silencing Congenital muscular dystrophy COL1A1 AAV VECTOR Muscular dystrophy MD Joint laxity Biological sciences Skeletal muscle