Comparison of Reporting Quality in National Cystic Fibrosis Patient Registries: Implications for Identifying Use of Novel CFTR Modulators

Comparison of Reporting Quality in National Cystic Fibrosis Patient Registries: Implications for Identifying Use of Novel CFTR Modulators

Abstract Introduction Advances in development of cystic fibrosis transmembrane conductance regulator modulator (CFTRm) therapies mean that now people who are heterozygous (instead of having to be homozygous) for the common F508del variant can benefit from these therapies. Recent economic estimates s...

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Bibliographic Details
Main Authors: Owen W. Tomlinson, Philip Mitchelmore, Craig A. Williams
Format: Article
Language:English
Published: Adis, Springer Healthcare 2024-09-01
Series:Pulmonary Therapy
Subjects:
Modulator therapy
Registry
Genotype
Respiratory disease
Ivacaftor
Lumacaftor
Online Access:https://doi.org/10.1007/s41030-024-00274-y
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https://doi.org/10.1007/s41030-024-00274-y

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