Overview

Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)

Status:
Recruiting

Trial end date:
2028-06-01

Target enrollment:

Participant gender:

Summary

The primary purpose of this study is to evaluate the safety and tolerability of ION356.

Phase:

Phase 1

Details

Lead Sponsor:

Ionis Pharmaceuticals, Inc.