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Gene Replacement Therapy for SMA Management

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As gene therapies emerge, clinicians need to understand how they work and associated broader implications for disease management. This journal supplement reviews the mechanisms by which viral vector systems correct gene defects and corresponding protein deficits, and discusses findings from recent and ongoing clinical trials.

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Video | Pulmonary | CME (AMA PRA Category 1 Credit) | 0.75 - 45 mins | Gastroenterology | Genetic Counselor | Internal Medicine | Neurology | Neuromuscular Specialist | Pediatrics | Primer/Supplements | Nurse | Nurse Practitioner | Ob/Gyn & Women's Health | Physical Medicine & Rehabilitation | Spinal Muscular Atrophy | Genomic Medicine | Neonatal/Perinatal  | Neurosurgery | Orthopedic Surgery |