A Therapy for Most with Cystic Fibrosis.
Cell
; 180(2): 211, 2020 01 23.
Article
em En
| MEDLINE
| ID: mdl-31978337
ABSTRACT
TRIKAFTA is the third drug approved by the FDA that rescues defects caused by the major mutation F508del. It is superior to its predecessors that were approved for patients who are homozygous for F508del because TRIKAFTA is also effective in CF patients who harbor only one copy of this mutation.
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Pirazóis
/
Piridinas
/
Quinolinas
/
Regulador de Condutância Transmembrana em Fibrose Cística
/
Fibrose Cística
/
Benzodioxóis
/
Aminofenóis
/
Indóis
Limite:
Humans
Idioma:
En
Ano de publicação:
2020
Tipo de documento:
Article