Overview

An Open-label Safety, Tolerability, and Dose-range Finding Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA)

Status:
Completed
Trial end date:
2013-01-31
Target enrollment:
Participant gender:
Summary
This objectives of this study are to evaluate the safety, tolerability, and pharmacokinetics of a single dose of nusinersen (ISIS 396443) administered intrathecally to participants with Spinal Muscular Atrophy (SMA).
Phase:
Phase 1
Details
Lead Sponsor:
Biogen
Ionis Pharmaceuticals, Inc.