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Pelizaeus-Merzbacher Disease
Philadelphia, Pennsylvania

Pelizaeus-Merzbacher Disease Clinical Trials in Philadelphia, Pennsylvania

3 recruitingPhiladelphia, Pennsylvania

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Showing 1–3 of 3 trials

Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher Disease

Pelizaeus-Merzbacher Disease

Ionis Pharmaceuticals, Inc.32 enrolled9 locationsNCT05659901

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

Pelizaeus-Merzbacher Disease

Ionis Pharmaceuticals, Inc.24 enrolled7 locationsNCT06150716

The Myelin Disorders Biorepository Project

AdrenoleukodystrophyCockayne SyndromeRefsum Disease+63 more

Children's Hospital of Philadelphia12,000 enrolled23 locationsNCT03047369

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