Gathering real-world compassionate data to expand eligibility to elexacaftor-tezacaftor-ivacaftor in people with cystic fibrosis with N1303K or other rare CFTR variants: a viewpoint.

Pierre Regis Burgel Isabelle Sermet-Gaudelus Emmanuelle GirodonReem KanaanJean Le BihanNatascha RemusBruno RavoninjatovoDominique GrenetMichele PorzioVéronique HoudouinLaurence Le Clainche-VialaIsabelle DurieuRaphaele Nove-JosserandJeanne LanguepinBérangère ColteyAnne GuillaumotCamille AudoussetRaphaël ChironLaurence WeissIsabelle FajacJennifer Da SilvaClémance Martinnull null

Published in: The European respiratory journal (2024)

Keyphrases

cystic fibrosis
electronic health record
copy number
big data
gene expression
artificial intelligence