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Pelizaeus-Merzbacher Disease clinical trials in Pennsylvania
4 studies for Pelizaeus-Merzbacher Disease are looking for participants at sites in Pennsylvania.
Cities in Pennsylvania
Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher Disease
- Ionis Investigative Site, Philadelphia
RecruitingObservational study
6 Months – 17 Years · Men · Updated Oct 2, 2026 · NCT05659901Longitudinal Study of Neurodegenerative Disorders
- UPMC Children's Hospital of Pittsburgh, Pittsburgh
RecruitingObservational study
All ages · All sexes · Updated Feb 9, 2026 · NCT03333200Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)
- Ionis Investigative Site, Philadelphia
RecruitingPhase 1
Ages 2 to 17 · Men · Updated Dec 12, 2025 · NCT06150716The Myelin Disorders Biorepository Project
- The Children's Hospital of Philadelphia, Philadelphia
- University of Pennsylvania, Philadelphia
- University of Pittsburgh Medical Center, Pittsburgh
RecruitingObservational study
All ages · All sexes · Updated Oct 23, 2025 · NCT03047369From ClinicalTrials.gov, data retrieved Oct 6, 2026. Each study sets its own eligibility; the study team decides who can join.