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Updated: Jul 15, 2025

Genetic Analysis of Hereditary Transthyretin Ala97Ser Related Amyloidosis
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遺伝性トランスチレチンアミロイドーシスとポリネーロパシーに対するEplontersen

Teresa Coelho1, Wilson Marques2, Noel R Dasgupta3

  • 1Centro Hospitalar Universitário de Santo António, Porto, Portugal.

JAMA
|September 28, 2023
PubMed
まとめ

Eplontersenは血清トランスチレチンの濃度を大幅に低下させ,遺伝性トランスチレチンアミロイド多神経症患者の神経症と生活の質を改善しました. この遺伝子静止治療は 新しい治療法として有望です

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