Niemann-pick Disease
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CLINICAL_TRIALS trial_result · 2026-02-19Clinical Trial Update: PREDIGA 2: Spanish Acronym of "Educational and Diagnostic Project for Gaucher and ASMD"
Clinical trial NCT05641103 (PREDIGA 2), an Educational and Diagnostic Project for Gaucher disease and acid sphingomyelinase deficiency, has …
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CLINICAL_TRIALS trial_result · 2015-07-30Clinical Trial Update: Tolerability and Safety Study of Recombinant Human Acid Sphingomyelinase in Acid Sphingomyelinase Deficiency Patients [Phase 1]
Phase 1 trial evaluating tolerability and safety of recombinant human acid sphingomyelinase in acid sphingomyelinase deficiency patients has…
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CLINICAL_TRIALS trial_result · 2015-03-19Clinical Trial Update: Safety Study of rhASM Enzyme Replacement Therapy in Adults With Acid Sphingomyelinase Deficiency (Niemann-Pick Disease) [Phase 1]
Phase 1 safety study of recombinant human acid sphingomyelinase enzyme replacement therapy for adults with Niemann-Pick disease was terminat…