23 studies found for:    "niemann-pick disease"
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Rank Status Study
21 Recruiting Molecular and Cellular Mechanisms of Lysosomal Storage Diseases
Condition: Lysosomal Storage Disorders
Intervention:
22 Recruiting Trial in Children With Growth Failure Due to Early Onset Lysosomal Acid Lipase (LAL) Deficiency/Wolman Disease
Conditions: Lysosomal Acid Lipase Deficiency;   Wolman Disease
Intervention: Drug: Sebelipase alfa (SBC-102)
23 Terminated Children With Lysosomal Acid Lipase Deficiency Who Previously Received Treatment With SBC-102
Conditions: Lysosomal Acid Lipase Deficiency;   Wolman Disease
Intervention: Drug: SBC-102

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