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A Long-term Follow-up Study of Gaucher Disease
The study aims are to: a) identify the long-term natural history of Gaucher disease, b) evaluate long-term treatment efficacy of enzyme replacement therapy (ER…
Gaucher Disease
Duke UniversityNCT03190837
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