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