Presented by Claudia A. Chiriboga, MD, MPH, FAAN and Tina Duong, PT, PhD

Supported through a grant from Novartis Gene Therapies.

Spinal Muscular Atrophy (SMA), one of the most common autosomal recessive diseases, is a neuromuscular disorder characterized by degeneration of spinal motor neurons resulting in variable degrees of muscular wasting and weakness. Among the many symptom presentations, depending on phenotype, are limited movement, trouble breathing, feeding, and swallowing.

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