Pelizaeus-merzbacher Disease
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CLINICAL_TRIALS research · 2026-04-17Clinical Trial Update: Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher Disease
Ionis Pharmaceuticals is conducting a study to characterize biomarkers and disease progression in Pelizaeus-Merzbacher Disease. The trial st…
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CLINICAL_TRIALS research · 2025-12-12Clinical Trial Update: Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD) [Phase 1]
A Phase 1 clinical trial evaluating the safety, pharmacokinetics, and pharmacodynamics of intrathecally administered ION356 in participants …
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CLINICAL_TRIALS research · 2025-10-23Clinical Trial Update: The Myelin Disorders Biorepository Project
This clinical trials update describes the Myelin Disorders Biorepository Project (NCT03047369) from Children’s Hospital of Philadelphia, cur…
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CLINICAL_TRIALS research · 2025-09-08Clinical Trial Update: UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells [Phase 1]
Phase 1 trial evaluating intrathecal umbilical cord blood-derived oligodendrocyte-like cells for several inherited metabolic brain diseases;…
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CLINICAL_TRIALS trial_result · 2016-05-13Clinical Trial Update: Long-Term Follow-Up Study of Human Stem Cells Transplanted in Subjects With Connatal Pelizaeus-Merzbacher Disease (PMD)
This document reports the long-term follow-up of subjects who received human stem cell transplants for Pelizaeus-Merzbacher disease; the tri…
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CLINICAL_TRIALS trial_result · 2015-01-15Clinical Trial Update: Study of Human Central Nervous System (CNS) Stem Cells Transplantation in Pelizaeus-Merzbacher Disease (PMD) Subjects [Phase 1]
This update reports completion of a Phase 1 study evaluating transplantation of human central nervous system stem cells in patients with Pel…