TrialJournal of inherited metabolic disease2026

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.

Melissa P Wasserstein et al.PubMed ↗Full text ↗Publisher ↗

No numbers read from the abstract.