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Updated: May 5, 2026

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ファブリー病の女性におけるミガラスタットの長期有効性

Staci Kallish1, Antonia Camporeale2, Robert J Hopkin3,4

  • 1Division of Translational Medicine and Human Genetics, Department of Medicine, The Perelman School of Medicine at the University of Pennsylvania, Philadelphia, Pennsylvania, USA staci.kallish@pennmedicine.upenn.edu.

Journal of medical genetics
|September 2, 2025
PubMed
まとめ

ミガラスタットは,進行性疾患であるファブリー病の女性に対する長期的な有効性を示しています. この研究は,女性における重大な疾患負担を強調し,治療の選択肢としてミガラスタットを支持しています.

キーワード:
心血管疾患ファブリー病遺伝的疾患,X関連遺伝学変異

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