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