Improvement in quality of life and general functions in pediatric acid sphingomyelinase deficiency patients after receiving olipudase alfa: A single-center experience in Taiwan.

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Title: Improvement in quality of life and general functions in pediatric acid sphingomyelinase deficiency patients after receiving olipudase alfa: A single-center experience in Taiwan.
Authors: Pan YW; Department of Pediatrics, National Cheng Kung University Hospital, College of Medicine, No. 138, Shengli Rd., North Dist., Tainan City, Taiwan., Yu WH; Department of Pediatrics, National Cheng Kung University Hospital, College of Medicine, No. 138, Shengli Rd., North Dist., Tainan City, Taiwan., Tsai MC; Department of Pediatrics, National Cheng Kung University Hospital, College of Medicine, No. 138, Shengli Rd., North Dist., Tainan City, Taiwan., Chou YY; Department of Pediatrics, National Cheng Kung University Hospital, College of Medicine, No. 138, Shengli Rd., North Dist., Tainan City, Taiwan.
Source: Molecular genetics and metabolism reports [Mol Genet Metab Rep] 2026 Apr 17; Vol. 47, pp. 101312. Date of Electronic Publication: 2026 Apr 17 (Print Publication: 2026).
Publication Type: Journal Article
Journal Info: Publisher: Elsevier Inc Country of Publication: United States NLM ID: 101624422 Publication Model: eCollection Cited Medium: Print ISSN: 2214-4269 (Print) Linking ISSN: 22144269 NLM ISO Abbreviation: Mol Genet Metab Rep Subsets: PubMed not MEDLINE
Database: MEDLINE Ultimate
Description
ISSN:2214-4269
DOI:10.1016/j.ymgmr.2026.101312