{"id":410481,"date":"2025-11-20T00:30:00","date_gmt":"2025-11-19T23:30:00","guid":{"rendered":"https:\/\/medizinonline.com\/?p=410481"},"modified":"2025-10-23T14:43:41","modified_gmt":"2025-10-23T12:43:41","slug":"donnees-a-long-terme-sur-les-modulateurs-de-cftr-chez-les-enfants-atteints-de-mucoviscidose","status":"publish","type":"post","link":"https:\/\/medizinonline.com\/fr\/donnees-a-long-terme-sur-les-modulateurs-de-cftr-chez-les-enfants-atteints-de-mucoviscidose\/","title":{"rendered":"Donn\u00e9es \u00e0 long terme sur les modulateurs de CFTR chez les enfants atteints de mucoviscidose"},"content":{"rendered":"\n

La mucoviscidose, \u00e9galement appel\u00e9e fibrose kystique (CF), est l’une des maladies m\u00e9taboliques cong\u00e9nitales les plus courantes en Europe. Les options th\u00e9rapeutiques pour les personnes atteintes de mucoviscidose se sont consid\u00e9rablement \u00e9largies au cours des derni\u00e8res ann\u00e9es. Les modulateurs de CFTR am\u00e9liorent la fonction de la prot\u00e9ine CFTR d\u00e9fectueuse et sont donc les premiers m\u00e9dicaments \u00e0 avoir une action causale, du moins partielle, dans la mucoviscidose. Une \u00e9tude d’extension de phase IIIb en open label a \u00e9valu\u00e9 l’utilisation d’une th\u00e9rapie combin\u00e9e \u00e0 base d’ivacaftor, d’elexacaftor et de tezacaftor chez des enfants \u00e2g\u00e9s de 6 \u00e0 11 ans sur une p\u00e9riode de 96 semaines. <\/strong><\/p>\n\n

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La mucoviscidose, \u00e9galement appel\u00e9e fibrose kystique (CF), est l’une des maladies m\u00e9taboliques cong\u00e9nitales les plus courantes en Europe. Les options th\u00e9rapeutiques pour les personnes atteintes de mucoviscidose se sont consid\u00e9rablement……... <\/p>\n

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