HPS-144 - Real-world experience of Avalglucosidase Alfa in infantile-Onset Pompe Disease: A case series study

• At: Copenhagen (Denmark) (2025)
• Type: Poster
• Poster code: HPS-144
• By: CHENG, Ya Ting (Kaohsiung Medical University Gangshan Hospital, China Taiwan)
• Co-author(s): Ms Ya Ting Cheng (Kaohsiung Medical University Gangshan Hospital, Kaohsiung, China Taiwan)
  Ms Wun-Syuan Chen (Kaohsiung Medical University Gangshan Hospital, Kaohsiung, China Taiwan)
  Ms Wei-Hsuan Huang (Kaohsiung Medical University Gangshan Hospital, Kaohsiung, China Taiwan)
  Ms Hsiu-Mei Chang (Kaohsiung Medical University Gangshan Hospital, Kaohsiung, China Taiwan)
  
• Abstract:

  Introduction
  Pompe disease is a rare, incurable hereditary condition caused by mutations in the GAA gene, which provides the instructions for making the enzyme alpha-glucosidase. Deficiency of alpha-glucosidase can lead to the accumulation of sugar to toxic levels in lysosomes. The buildup of sugar harms organs throughout the body particularly..

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