P034: A real-world evidence study into the usage of olipudase alfa for acid sphingomyelinase deficiency in the United States

Genetics in Medicine Open 2026 Open access

Read full text
Authors: Judy Hull, Neeraj Singh Rawat, Laurence Pollissard, Gavin Harper, Joe Conyers, Rabiyah Sahar
DOI: 10.1016/j.gimo.2026.103525
Cited by: 0

Abstract

Acid sphingomyelinase deficiency (ASMD) is a rare, progressive lysosomal storage disorder with diverse clinical manifestations. Olipudase alfa is an enzyme replacement therapy approved for non-central nervous manifestations of ASMD in pediatric and adult patients. This study evaluated the factors affecting olipudase alfa treatment initiation among patients with ASMD type B and type A/B in the United States.

Fields of Study & Subjects

Lysosomal Storage Disorders Research Metabolism and Genetic Disorders Glycogen Storage Diseases and Myoclonus

Publication details

Journal / source
Genetics in Medicine Open
Volume
4
Pages
103525–103525
Publication date
1 January 2026
Language
EN