Other innovative therapies in Duchenne muscular dystrophy
1Evolution of Neuromuscular Diseases - Innovative Concepts and Practice (END-ICAP) U1179, Université Paris-Saclay, Université de Versailles Saint-Quentin-en-Yvelines, Inserm, 78000 Versailles, France; Pediatric Department, Raymond Poincaré Hospital, Université Paris-Saclay, Université de Versailles Saint-Quentin-en-Yvelines, Assistance Publique Hôpitaux de Paris, 92380 Garches, France.
Abstract:
Over the past three decades, new therapeutic strategies have been developed to treat Duchenne muscular dystrophy (DMD). These strategies aim to correct the primary genetic defect, compensate for secondary pathological changes resulting from muscular dystrophy, or stimulate skeletal muscle growth and regeneration to overcome muscle wasting. This article discusses three concepts currently being tested on patients with DMD: strategies for restoring dystrophin, next-generation pharmacological agents, and cell therapy.
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