Acid Sphingomyelinase Deficiency
📧 Sign up to get email alerts when something new happens for Acid Sphingomyelinase Deficiency.
Sign up freeTop drugs studied for this disease
-
CLINICAL_TRIALS trial_result · 2026-04-13Clinical 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 · 2026-02-19Clinical 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 …
-
CLINICAL_TRIALS research · 2026-02-11Clinical Trial Update: GammaGA: Prevalence of Acid Sphingomyelinase Deficiency Disease (ASMD) and Gaucher Disease in Patients With Monoclonal Gammopathies and/or Multiple Myeloma
Clinical trial update for GammaGA examining how common acid sphingomyelinase deficiency (ASMD) and Gaucher disease are in patients with mono…
-
EMA approval · 2026-01-19EMA Authorised: Xenpozyme (olipudase alfa) — Sanofi B.V.
The EMA authorised Xenpozyme (olipudase alfa) for the treatment of Acid sphingomyelinase deficiency (ASMD) types A/B or type B. It has been …
-
CLINICAL_TRIALS research · 2025-12-10Clinical Trial Update: Diagnostic Creteria of Acid Sphingomyelinase Deficiency (ASMD)
The entry reports an update to the diagnostic criteria for Acid Sphingomyelinase Deficiency (ASMD) in an active, non-recruiting clinical tri…
- Want full summaries, importance scores, and drug timelines? Upgrade to Plus or Pro.
-
CLINICAL_TRIALS research · 2025-10-31Clinical Trial Update: A Real-world Long-term Safety and Immunogenicity Study of Olipudase Alfa Therapy in Pediatric Patients Less Than 2 Years of Age With Acid Sphingomyelinase Deficiency (ASMD)
This is an ongoing real-world, long-term safety and immunogenicity study of olipudase alfa in infants under 2 years with acid sphingomyelina…
-
CLINICAL_TRIALS research · 2025-09-23Clinical Trial Update: A Non-Interventional National Study in Pediatric Patients With Unexplained Enlarged Spleen
A non-interventional national pediatric study sponsored by Sanofi investigating unexplained enlarged spleen in patients with Gaucher disease…
-
CLINICAL_TRIALS research · 2025-05-22Clinical Trial Update: National Multicentre Study of the Natural History of Acid Sphingo-myelinase Deficiency in Adults and Children
A national multicentre observational study investigating the natural history of acid sphingomyelinase deficiency (ASMD) in adults and childr…
-
CLINICAL_TRIALS trial_result · 2025-02-06Clinical 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…
-
FDA approval · 2024-12-20FDA Approved: XENPOZYME — Supplemental Application (Genzyme Corporation)
FDA approved a supplemental application for XENPOZYME (olipudase alfa-RPCP) from Genzyme Corporation.
-
CLINICAL_TRIALS trial_result · 2024-07-03Clinical 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…
-
FDA approval · 2023-12-05FDA Approved: XENPOZYME — Supplemental Application (Genzyme Corporation)
FDA approved a supplemental application for XENPOZYME (olipudase alfa-rpcp) from Genzyme Corporation. This supplemental approval concerns th…
-
FDA approval · 2022-08-31FDA Approved: XENPOZYME — Original Application (Genzyme Corporation)
FDA approved XENPOZYME (olipudase alfa-rpcp) from Genzyme in an original application to treat acid sphingomyelinase deficiency (ASMD), inclu…
-
CLINICAL_TRIALS trial_result · 2022-05-24Clinical 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…
-
CLINICAL_TRIALS trial_result · 2021-02-01Clinical 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 · 2015-07-30Clinical 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…
-
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…