HPS-143 - Effectiveness and safety of idursulfase enzyme replacement therapy in infants diagnosed with Mucopolysaccharidosis II through newborn screening: A case series

• At: Copenhagen (Denmark) (2025)
• Type: Poster
• Poster code: HPS-143
• By: CHEN, Wun-Syuan (Department of Pharmacy, Kaohsiung Medical University Gangshan Hospital, China Taiwan)
• Co-author(s): Ms Wun-Syuan Chen (Department of Pharmacy, Kaohsiung Medical University Gangshan Hospital, Kaohsiung City, China Taiwan)
  Ms Ya-Ting Cheng (Department of Pharmacy, Kaohsiung Medical University Gangshan Hospital, Kaohsiung City, China Taiwan)
  Ms Wei-Hsuan Huang (Department of Pharmacy, Kaohsiung Medical University Gangshan Hospital, Kaohsiung City, China Taiwan)
  Mrs Hsiu-Mei Chang (Department of Pharmacy, Kaohsiung Medical University Gangshan Hospital, Kaohsiung City, China Taiwan)
  
• Abstract:

  Introduction:
  Mucopolysaccharidosis type Ⅱ (MPS Ⅱ, Hunter syndrome) is a rare X-linked recessive lysosomal storage disorder caused by deficiency of iduronate 2-sulfatase (IDS), resulting in accumulation of glycosaminoglycans (GAGs) in multiple organ systems. Heparan sulfate and dermatan sulfate are members of GAG family, which are implicated..

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