The Genetic Disorders Of Mucociliary Clearance Consortium is a network of nine North American Centers that are collaborating in the diagnostic testing, genetic studies, and clinical trials in patients with impairments in mucociliary clearance, focusing on primary ciliary dyskinesia, cystic fibrosis, and pseudohypoaldosteronism. Additionally, GDMCC studies target related clinical conditions believed to be due to impaired mucociliary clearance including idiopathic bronchiectasis and infection with non-tuberculous mycobacterial (NTM) organisms. Ultimately, we hope to better define the clinical pathogenesis of these important airway diseases, improve or expand diagnostic testing, and develop new and effective treatments.
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 and comparative clinical studies in cystic fibrosis (CF), pseudohypoaldosteronism (PHA) and other chronic airway disease.
The consortium currently consists of nine primary sites:
Lead site: University of North Carolina at Chapel Hill (UNC)
- Washington University in St. Louis (Missouri)
- University of Washington (Seattle, Washington)
- University of Colorado, Denver Children's Hospital (Denver, Colorado)
- The Hospital for Sick Children (Toronto, Ontario, Canada)
- Stanford University (Palo Alto, California)
- National Institute for Allergy and Infectious Diseases (Bethesda, MD)
- St. Michael's Hospital (Toronto, Ontario, Canada)
- National Jewish Health (Denver, Colorado)
All GDMCC sites have expertise in studying the genetics and pathogenesis of airways disease, 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.
The goals of this consortium is are to:
- Improve diagnostic testing for unusual but important, inherited airway diseases, including primary ciliary dyskinesia (PCD), atypical forms of cystic fibrosis (CF), and pseudohypoaldosteronism (PHA).
- Better understand how these and related disorders like idiopathic bronchiectasis and NTM pulmonary disease cause progressive lung involvement.
- Develop new and effective treatments for these diseases.