Case report: Treatment of the severe ASMD phenotype with olipudase alfa and JZP-150

Molecular Genetics and Metabolism 2026

View at publisher

Authors: Justin Hopkin, Steven Laffoon, Joy Chen, Sophia C. Abner

DOI: 10.1016/j.ymgme.2025.109468

Cited by: 0

Fields of Study & Subjects

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