Therapies Used by Children With Primary Ciliary Dyskinesia: A Natural History Study
Older participants and those with inner dynein arm/microtubular disorganization defects reported more therapies likely due to disease progression and more severe phenotypes, respectively. We report that a wide range of therapies are used in PCD without disease-specific studies defining benefits and risks.
Authors
- Margaret W. Leigh (ORCID: https://orcid.org/0000-0002-6420-2459)
- Lang Li (ORCID: https://orcid.org/0000-0002-6479-5952)
- Sharon Dell (ORCID: https://orcid.org/0000-0003-2169-9407)
- Maimoona A. Zariwala (ORCID: https://orcid.org/0000-0003-1619-1393)
- Kelli M. Sullivan (ORCID: https://orcid.org/0000-0001-8564-4726)
- Michael R. Knowles (ORCID: https://orcid.org/0000-0002-4672-5969)
- Stephanie D. Davis (ORCID: https://orcid.org/0000-0002-9390-0651)
- Scott D. Sagel (ORCID: https://orcid.org/0000-0001-6172-4465)
- Robert A. Gardner (ORCID: https://orcid.org/0000-0002-9123-0762)
- Margaret Rosenfeld (ORCID: https://orcid.org/0000-0002-8105-614X)
- Carlos Milla (ORCID: https://orcid.org/0000-0001-5515-3053)
- Feng‐Chang Lin (ORCID: https://orcid.org/0000-0002-2638-1775)
- Thomas Ferkol
Institutions
- University of North Carolina at Chapel Hill (US)
- University of British Columbia (CA)
- Palo Alto University (US)
- University of Washington (US)
- Lung Institute (US)
- Levine Children's Hospital (US)
- Wake Forest University (US)
- University of Colorado Anschutz Medical Campus (US)
- University of Colorado Denver (US)
- Stanford University (US)
Publication Details
- Journal
- Pediatric Pulmonology
- Published
- 2024-11-22
- DOI
- https://doi.org/10.1002/ppul.27412
- Citations
- 3
- Primary Topic
- Cystic Fibrosis Research Advances
- Type
- article
- Field-Weighted Citation Impact
- 1.26
Funders
- Yale University
- Georgia Clinical and Translational Science Alliance
- Rare Diseases Clinical Research Network
- National Institutes of Health
- National Heart, Lung, and Blood Institute
- National Center for Advancing Translational Sciences