The mission of the GDMCC is to create and maintain a clinical research network to study rare diseases of the airways, focusing on primary ciliary dyskinesia (PCD); NTM pulmonary disease and idiopathic bronchiectasis with rigorous diagnostic evaluations; comparative clinical studies in cystic fibrosis (CF); pseudohypoaldosteronism (PHA); and other chronic airway diseases.
The consortium currently consists of nine primary sites:
Lead site: University of North Carolina at Chapel Hill (UNC)
- Washington University in St. Louis (St. Louis, Missouri)
- University of Washington (Seattle, Washington)
- University of Colorado, Denver Children's Hospital (Denver, Colorado)
- Stanford University (Palo Alto, California)
- National Heart, Lung and Blood Institute (Bethesda, Maryland)
- National Jewish Health (Denver, Colorado)
- Indiana University (Indianapolis, Indiana)
- The Hospital for Sick Children (Toronto, Ontario, Canada)
All GDMCC sites have expertise in studying the genetics and pathogenesis of airway diseases, design of clinical trials, and development of novel therapeutics for defective lung defenses. In this proposal, we will develop collaborative, intellectual, and methodological resources for the study of rare diseases of the airways. Finally, the consortium plans to establish training programs in rare airways diseases, and develop regional and internet-based networks to provide information about such rare diseases to the patients, medical professionals, and lay public for education, referral, and recruitment of study subjects.