GlobalResearch and clinical trials
Olipudase alfa in adults with ASMD: spleen and liver shrinkage continued over up to five years
A study published in a journal and reviewed by independent scientists. Even so, a single study is not conclusive; it needs to be replicated.
Final results for the enzyme therapy olipudase alfa in adults with acid sphingomyelinase deficiency have been published. Of the 36 adults in the ASCEND trial, 35 continued treatment for up to five years. The lungs' gas transfer capacity increased by an average of 35.9% from baseline. Spleen volume decreased by an average of 57.5%, and liver volume by 36.8%. No new safety issues were seen.
What does it mean for patients and families?
This treatment is not for symptoms affecting the brain. Access in Türkiye depends on TİTCK (Turkish Medicines and Medical Devices Agency) and SGK (Social Security Institution) processes; treatment decisions are made with a metabolic disease specialist.
Source
Scientific paper: Adults With Acid Sphingomyelinase Deficiency Have Sustained Improvements in Clinical Outcomes With up to 5 Years of Olipudase Alfa Enzyme Replacement Therapy: ASCEND Trial Final Results. Journal of Inherited Metabolic Disease, 2026. DOI: 10.1002/jimd.70192 · PubMed 42047222
The source page was opened and read on October 7, 2026. Summary prepared by the Doktorclub editorial team and translated from Turkish; it has not yet been reviewed by a physician (a scientific board is being set up).
This page is translated from the Turkish original.
Related disease: Acid sphingomyelinase deficiency (ASMD, Niemann-Pick disease types A/B)
This page reports a development; it is not a treatment recommendation. Your doctor decides whether a treatment is right for you.