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How Gene Replacement Therapy is Changing the Way We Manage Spinal Muscular Atrophy and Other Neurologic Diseases

How Gene Replacement Therapy is Changing the Way We Manage Spinal Muscular Atrophy and Other Neurologic Diseases

Format: Supplement
Credits: 1.0 AMA PRA Category 1 Credit™

Innovations in technology, delivery, and vector systems in gene therapy mean that patients with potentially devastating neuromuscular disorders, such as spinal muscular atrophy (SMA), are now candidates for treatment.  As these therapies emerge, clinicians need to understand how they work and associated broader implications for disease management. This 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. In addition, the authors—all experts in SMA—provide commentary on related topics and respond to frequently asked questions about SMA and gene replacement therapy.

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