Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)
for Pelizaeus-Merzbacher Disease
3 locations incl. Atlanta
Sponsor: Ionis Pharmaceuticals, Inc.
Sex: Male·Age: 2 - 17
Code: NCT06150716·Phase 1·Recruiting