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

~Estimated

~$10 – $8,750

Calico Life Sciences LLCNCT05757141

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

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

~Estimated

~$10 – $6,250

University of PittsburghNCT03333200

~Estimated

~$10 – $750

Children's Hospital of PhiladelphiaNCT03047369