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...
Saved in:
| 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: | |
| Online Access: | https://doi.org/10.1007/s41030-024-00274-y |
| Tags: |
Add Tag
No Tags, Be the first to tag this record!
|
Similar Items
-
Advances in the treatment of cystic fibrosis: CFTR modulators
by: Maria Dolores Pastor-Vivero, et al.
Published: (2025-05-01) -
Progress of personalized medicine of cystic fibrosis in the times of efficient CFTR modulators
by: Burkhard Tümmler, et al.
Published: (2025-05-01) -
Analysis of Ivacaftor drug approval for cystic fibrosis patients with gating mutations.
by: Nemirajaiah, S.
Published: (2021-08-01) -
Blood platelet reduction after elexacaftor/tezacaftor/ivacaftor treatment in people with cystic fibrosis may depend on systemic inflammation reduction
by: Alice Castaldo, et al.
Published: (2025-07-01) -
Impact of CFTR Modulators on Longitudinal Cystic Fibrosis Survival and Mortality: Review and Secondary Analysis
by: Jaime L. Rubin, et al.
Published: (2025-07-01)