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