Overview

A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Participants With Later-onset Spinal Muscular Atrophy (SMA)

Status:
Completed
Trial end date:
2017-02-20
Target enrollment:
Participant gender:
Summary
The primary objective of this study is to examine the clinical efficacy of nusinersen (ISIS 396443) administered intrathecally to participants with later-onset Spinal Muscular Atrophy (SMA). The secondary objective is to examine the safety and tolerability of nusinersen administered intrathecally to participants with later-onset SMA.
Phase:
Phase 3
Details
Lead Sponsor:
Biogen
Ionis Pharmaceuticals, Inc.