Adult Spinal Muscular Atrophy Clinical Trial
Official title:
Effect of Nusinersen on Motor Function in Adult Patients With Spinal Muscular Atrophy Types 2 and 3
Observational study of adult patients with spinal muscular atrophy types 2 and 3 receiving nusinersen
Observational study to assess effects of nusinersen on motor function in adult patients with spinal muscular atrophy who are both ambulatory and non-ambulatory. Subjects will receive standard of care with nusinersen intrathecal injection and undergo baseline and every 6 month motor assessments and pulmonary function testing during the first two years of treatment with nusinersen. ;