Additional file 1 of Metabolic improvement in patients with acid sphingomyelinase deficiency following intravenous trehalose administration: an untargeted pharmacometabolomic study
Fields of Study & Subjects
Lysosomal Storage Disorders Research
Metabolism and Genetic Disorders
Sphingolipid Metabolism and Signaling
Publication details
- Journal / source
- Figshare
- Publication date
- 1 January 2026