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Updated: Sep 9, 2025

In Vitro Enzyme Measurement to Test Pharmacological Chaperone Responsiveness in Fabry and Pompe Disease
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米国で酵素置換療法を受けた患者のポンペ病の経済的負担に関する遡及コホート研究

Robert D Steiner1, Tmirah Haselkorn2, Nishitha R Pillai3

  • 1Department of Pediatrics, University of Wisconsin School of Medicine and Public Health, Madison, WI, USA.

Journal of medical economics
|September 4, 2025
PubMed
まとめ

ポンペ病は酵素置換療法 (ERT) を受けている患者に重大な経済的負担を課します. 費用は主に門診サービスとERT自体に左右され,幼児発症のポンペ病 (IOPD) と遅発症のポンペ病 (LOPD) の間で変動します.

キーワード:
I10 についてI19 についてポンペ病経済的な負担酵素置換療法医療資源の利用現実の世界

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