HIGHLIGHTS
- who: Chung-Lin Lee and colleagues from the Department of Pediatrics, MacKay Memorial Hospital, Taipei, Taiwan Institute of Clinical Medicine, National Yang-Ming Chiao-Tung University, Taipei, Taiwan have published the research: Efficacy of Intravenous Elosulfase Alfa for Mucopolysaccharidosis Type IVA: A Systematic Review and Meta-Analysis, in the Journal: (JOURNAL)
- what: Our meta-analysis showed that ERT for MPS IVA has beneficial effects on uKS, physical performance, life quality, and respiratory function.
- how: (MPS or lysosomal storage disease with an autosomal recessive inherited pattern is induced by GALNS gene mutations causing deficiency . . .
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