Acid Sphingomyelinase Deficiency

• CLINICAL_TRIALS trial_result · 2026-04-13
  Clinical Trial Update: A Study to Evaluate Safety and Tolerability of Olipudase Alfa in Pediatric and Adult Participants With Acid Sphingomyelinase Deficiency (ASMD) Who Completed the DFI12712 or the LTS13632 Study in France [Phase 2]

  A Phase 2 trial evaluating safety and tolerability of olipudase alfa in pediatric and adult participants with acid sphingomyelinase deficien…

• CLINICAL_TRIALS trial_result · 2026-02-19
  Clinical 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 …

• CLINICAL_TRIALS trial_result · 2025-02-06
  Clinical Trial Update: Data Analysis of Adult and Pediatric Participants With Acid Sphingomyelinase Deficiency (ASMD) on Early Access to Olipudase Alfa in France

  This document reports a completed data analysis of adult and pediatric participants with acid sphingomyelinase deficiency (ASMD) who receive…

• CLINICAL_TRIALS trial_result · 2024-07-03
  Clinical Trial Update: A Long-Term Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency [Phase 2]

  This document reports long-term results from a Phase 2 study of olipudase alfa in patients with acid sphingomyelinase deficiency (Niemann-Pi…

• CLINICAL_TRIALS trial_result · 2022-05-24
  Clinical Trial Update: Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency [Phase 2, Phase 3]

  Phase 2/3 trials evaluated the efficacy, safety, pharmacodynamics, and pharmacokinetics of olipudase alfa in patients with acid sphingomyeli…

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• CLINICAL_TRIALS trial_result · 2021-02-01
  Clinical Trial Update: Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency [Phase 1, Phase 2]

  Phase 1/2 trial assessing repeat ascending doses of olipudase alfa in pediatric patients under 18 with acid sphingomyelinase deficiency; saf…

• CLINICAL_TRIALS trial_result · 2015-07-30
  Clinical 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…

• CLINICAL_TRIALS trial_result · 2015-03-19
  Clinical 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…