The Rare Lung Disease Consortium is designed to accelerate the development and application of new knowledge about rare lung disorders, and engage the respective lung disease communities and clinical research centers for treatment trials in rare lung diseases, with the ultimate goal of developing therapies that improve the quality of life for patients by driving the process from pilot studies to FDA approval. The Administrative Core will provide leadership and oversight to the network, building on the institutional support of major clinical research centers with CTSAs and a growing network of RLD clinical care sites. The leadership is comprised of scientists with international recognition for their research on the molecular pathogenesis, diagnosis, and therapy of rare lung disorders. The directors have significant experience in the organization, implementation, and effectiveness of a rare disease clinical research network providing a bridge between clinical investigators and trainees/academic institutions, patient groups, and the general public. The goals of the Administrative Core include coordinating and facilitating communications, organizing infrastructure support, managing policies and documentation, creating and distributing educational materials, and providing fiscal administration of the consortium. Project support includes the organization of a Biostatistics Core and Radiology Core, to provide standardized and expert support to the RLD clinical research studies. The Administrative Core will produce policy and procedure manuals to organize data management between and within the projects, and streamline IRB protocol approval to facilitate multicenter and multinational clinical research studies. The RLDC will collaborate with the RDCRC and the Data Management Coordination Center to collect and transfer data seamlessly between projects. The Administrative Core will also provide guidance on web portal content, and create web content as part of our training program. The RLCN directors and PIs have partnered with patient advocates to create a RLDC Steering Committee that will guide project and program strategies, with advice and oversight of internal and external experts in translational pulmonary research. Coordinated communication, standardized protocols, and administration of multi-center clinical trial sites will create an infrastructure to support significant advances in rare lung disease research.
The Rare Lung Disease Consortium will build a network of academic institutes, clinical! care centers, and patient advocates to create a research and training program that will make advances in the treatment of rare lung diseases. The Administrative Core will provide the leadership, communication strategies and financial administration to run the consortium smoothly, and create policies and procedures for human research protocols and data management that will provide standardized models for research across the network.
|Vicary, Glenn W; Vergne, Yeidyly; Santiago-Cornier, Alberto et al. (2016) Pulmonary Fibrosis in Hermansky-Pudlak Syndrome. Ann Am Thorac Soc 13:1839-1846|
|Sahin, Mustafa; Henske, Elizabeth P; Manning, Brendan D et al. (2016) Advances and Future Directions for Tuberous Sclerosis Complex Research: Recommendations From the 2015 Strategic Planning Conference. Pediatr Neurol 60:1-12|
|O'Brien, Kevin J; Lozier, Jay; Cullinane, Andrew R et al. (2016) Identification of a novel mutation in HPS6 in a patient with hemophilia B and oculocutaneous albinism. Mol Genet Metab 119:284-287|
|El-Chemaly, Souheil; Young, Lisa R (2016) Hermansky-Pudlak Syndrome. Clin Chest Med 37:505-11|
|Fan, Leland L; Dishop, Megan K; Galambos, Csaba et al. (2015) Diffuse Lung Disease in Biopsied Children 2 to 18 Years of Age. Application of the chILD Classification Scheme. Ann Am Thorac Soc 12:1498-505|
|Saito, Atsushi; Nikolaidis, Nikolaos M; Amlal, Hassane et al. (2015) Modeling pulmonary alveolar microlithiasis by epithelial deletion of the Npt2b sodium phosphate cotransporter reveals putative biomarkers and strategies for treatment. Sci Transl Med 7:313ra181|
|Trapnell, Bruce C (2015) A lymphocyte-mediated cause of secondary PAP. Blood 125:215-6|
|Suzuki, Takuji; Arumugam, Paritha; Sakagami, Takuro et al. (2014) Pulmonary macrophage transplantation therapy. Nature 514:450-4|