English

DTIC ADA563380: Columbia SMA Project: A Randomized, Control Trial of the Effects of Exercise on Motor Function and Strength in Patients with Spinal Muscular Atrophy (SMA)

This three-year randomized controlled trial is designed to assess the effectiveness of a comprehensive regimen incorporating strengthening and aerobic exercise in 14 ambulatory patients with spinal muscular atrophy (SMA). To date, 22 subjects have been screened and 10 subjects enrolled. Reasons for non-participation were most often cost and burden of travel. Protocol adherence has been outstanding thus far with only one deviation with regards to visit schedule. All testing and intervention procedures have been well tolerated. All non-serious adverse events are closely monitored by an independent safety monitor who has approved continuation of the study without modification thus far. The overall goal of this project is to provide novel insights into the effectiveness of aerobic and strengthening exercise to improve function in SMA, and explore the physiology underlying its effects.

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