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Acid Sphingomyelinase Deficiency clinical trials

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NCT ID: NCT00410566 Terminated - Clinical trials for Niemann-Pick Disease

Safety Study of rhASM Enzyme Replacement Therapy in Adults With Acid Sphingomyelinase Deficiency (Niemann-Pick Disease)

Start date: December 2006
Phase: Phase 1
Study type: Interventional

The purpose of this study is to determine the safe range of single doses of rhASM administered to adults with ASM deficiency.