Registry-based study in people with cystic fibrosis and an <i>R117H</i> variant treated with ivacaftor.

Higgins, Mark; Farietta, Thalia; Campbell, Daniel; Liu, Meng; Ostrenga, Josh; Elbert, Alexander; Shih, Judy; Volkova, Nataliya

Registry-based study in people with cystic fibrosis and an R117H variant treated with ivacaftor.

Higgins, Mark; Farietta, Thalia; Campbell, Daniel; Liu, Meng; Ostrenga, Josh; Elbert, Alexander; Shih, Judy; Volkova, Nataliya.

Afiliação

Higgins M; Vertex Pharmaceuticals Europe Ltd, London, UK Mark_Higgins@vrtx.com.
Farietta T; Vertex Pharmaceuticals Incorporated, Boston, Massachusetts, USA.
Campbell D; Vertex Pharmaceuticals Incorporated, Boston, Massachusetts, USA.
Liu M; Vertex Pharmaceuticals Incorporated, Boston, Massachusetts, USA.
Ostrenga J; Cystic Fibrosis Foundation, Bethesda, Maryland, USA.
Elbert A; Cystic Fibrosis Foundation, Bethesda, Maryland, USA.
Shih J; Vertex Pharmaceuticals Incorporated, Boston, Massachusetts, USA.
Volkova N; Vertex Pharmaceuticals Incorporated, Boston, Massachusetts, USA.

BMJ Open Respir Res ; 10(1)2023 05.

Article em En | MEDLINE | ID: mdl-37230763

Assuntos

Fibrose Cística; Adulto; Humanos; Criança; Fibrose Cística/tratamento farmacológico; Fibrose Cística/genética; Regulador de Condutância Transmembrana em Fibrose Cística/genética; Mutação; Sistema de Registros

Palavras-chave

Cystic Fibrosis

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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Fibrose Cística Tipo de estudo: Observational_studies / Prognostic_studies Limite: Adult / Child / Humans Idioma: En Ano de publicação: 2023 Tipo de documento: Article

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