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Systemic Delivery of MicroRNA Using Recombinant Adeno-associated Virus Serotype 9 to Treat Neuromuscular Diseases in Rodents
Published on: August 10, 2018
Neuromuscular diseases: Diagnosis and management
P Mary1, L Servais1, R Vialle1
1Service d'orthopédie et de chirurgie réparatrice de l'enfant, département hospitalo-universitaire maladies musculosquelettiques et innovations thérapeutiques (MAMUTH), hôpital d'enfants Armand-Trousseau, 26, avenue du Dr-A.-Netter, 75571 Paris cedex 12, France.
Pediatric orthopedic surgeons must understand neuromuscular diseases (NMDs) and their musculoskeletal manifestations. Early diagnosis and multidisciplinary care are crucial for optimal treatment timing and patient outcomes.
Area of Science:
- Orthopedics
- Neurology
- Pediatrics
Background:
- Neuromuscular diseases (NMDs) impact motor neurons, muscles, or the neuromuscular junction, presenting diverse syndromes.
- Key NMDs relevant to pediatric orthopedics include spinal muscular atrophy, Charcot-Marie-Tooth disease, muscular dystrophies, and myotonic dystrophy.
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