This is a longitudinal, observational study of ambulatory and non-ambulatory adult patients with genetically confirmed chromosome 5q spinal muscular atrophy (SMA) to examine the safety, tolerability, and effectiveness of SPINRAZA® (nusinersen) for up to 30 months.
This study does not provide SPINRAZA or cover costs associated with standard clinical care. These patients will be treated by their respective physicians according to standard clinical practice. Study visits include standardized assessments of strength and function, and occur at baseline, day 15, after treatment initiation, day 30, day 60, and then at 4-month intervals through month 30.
Principal Investigator: Shafeeq Ladha, MD