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Title   A Phase 2/3, Multicenter, Randomized, Double-blinded, Placebo-controlled, Repeat-dose Study to Evaluate the Efficacy, Safety, Pharmacodynamics, and Pharmacokinetics of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency
Disease/Condition   Brain and Neurological,Liver Diseases,Bones, Muscles & Joints (Orthopaedics),Genetic Disorders,Lung Diseases,Lymphatic and Spleen Diseases,Metabolic Diseases

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Eligibility Criteria   Click here for eligibility requirements
Principal Investigator   Melissa Wasserstein, MD
Contact Name   Joana Oliveira
Contact Email
Protocol Number   16-02-329-01
Inclusion/Notes   This information was obtained from
Status   Active/Enrolling
Adult Clinical Trial   Yes
Pediatric Clinical Trial   No
Sub-Specialty   Bones and Joints, Liver, Lung, Other Hematopoietic