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The Myelin Disorders Biorepository Project and Global Leukodystrophy Initiative Clinical Trials Network
The Myelin Disorders Biorepository Project (MDBP) seeks to collect and analyze clinical data and biological samples from leukodystrophy patients worldwide to s…
LeukodystrophyWhite Matter DiseaseLeukoencephalopathies+63 more
Children's Hospital of PhiladelphiaNCT03047369
Phase 1
A Phase 1b/2 Open-label Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Exploratory Efficacy Following Fosigotifator Administration in Adult and Pediatric Subjects With Vanishing White Matter Disease
Fosigotifator is an investigational drug being researched for the treatment of Vanishing White Matter disease in adult, pediatric and infant participants. This…
Vanishing White Matter Disease
Calico Life Sciences LLCNCT05757141
Longitudinal Study of Neurodegenerative Disorders
The purpose of this study is to understand the course of rare genetic disorders that affect the brain. This data is being analyzed to gain a better understandi…
MLDKrabbe DiseaseALD+24 more
University of PittsburghNCT03333200