A Study for Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in Nusinersen (ISIS 396443) Investigational Studies

Summary

The primary objective is to evaluate long-term safety and tolerability of nusinersen (ISIS 396443) administered by intrathecal (IT) injection to participants with Spinal Muscular Atrophy (SMA) who previously participated in investigational studies of nusinersen. The secondary objective is to examine the long-term efficacy of nusinersen administered by IT injection to participants with SMA who previously participated in investigational studies of nusinersen.

Timeline

Start

2015-11-04

Primary completion

2023-08-21

Completion

2023-08-21

Publications

• Kokaliaris C, Evans R, Hawkins N, Mahajan A, Scott DA, Sutherland CS, Nam J, Sajeev G. Long-Term Comparative Efficacy and Safety of Risdiplam and Nusinersen in Children with Type 1 Spinal Muscular Atrophy. Adv Ther. 2024 Jun;41(6):2414-2434. doi: 10.1007/s12325-024-02845-6. Epub 2024 May 5.

Drugs

Indications