- FDA Awards 11 Grants to Clinical Trials to Develop New Medical Products for Rare Disease Treatments | FDA
- FDA awards 6 grants to fund new clinical trials to advance the development of medical products for the treatment of rare diseases (October 8, 2020)
- FDA awards 12 grants to fund new clinical trials to advance the development of medical products for the treatment of rare diseases (October 8, 2019)
- FDA awards 2 grants for natural history studies in rare diseases (October 8, 2019)
- FDA orphan products clinical trial grants: assessment of outcomes and impacts on rare disease product development by Kathleen L. Miller, Christine Mueller, Gumei Liu, Katherine Needleman, Janet Maynard
- An analysis of follow-on development in new drug classes, January 1986-June 2018 by Michael L. Lanthier, Kirk W. Kerr and Kathleen L. Miller
- FDA’s Office of Orphan Products Development: providing incentives to promote the development of products for rare diseases by Soumya Patel and Katherine Needleman
- Precision Medicines’ Impact on Orphan Drug Designation by Christine M. Mueller, Gayatri R. Rao, and Katherine Needleman
- Dr. Kenneth Ataga at the University of Tennessee Health Science Center (UTHSC)
- Effect of renin-angiotensin-aldosterone system blocking agents on progression of glomerulopathy in sickle cell disease. British Journal of Haematology
- Plasma metabolomics analysis in sickle cell disease patients with albuminuria. British Journal of Haematology.
- Progressive decline in estimated GFR in patients with sickle cell disease: an observational cohort study. American Journal of Kidney Diseases.
- Rapid decline in estimated glomerular filtration rate is associated with mortality in adults with sickle cell disease. British Journal of Haematology.
- Jonathon Davis, MD at Tufts Medical Center - The role of recombinant human CC10 in the prevention of chronic pulmonary insufficiency of prematurity. Nature.
- Mrinal Gounder, MD at Memorial Sloan Kettering Cancer Center - Sorafenib for Advanced and Refractory Desmoid Tumors. New England Journal of Medicine.
Find More Orphan Buzz in the News:
- Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (RO1)
- Natural History Studies Addressing Unmet Needs of Rare Diseases: Orphan Products Research Project Grant (R01)
- RFA FD-15-001 for Receipt Date 2018
- RFA Natural History Studies for Rare Disease Product Development: Orphan Products Research Project Grant (R01)
- Notice of Cancellation of the Natural History October 2018 Receipt Date Past
- Federal Register for Clinical Studies RFA FD-11-001 For Receipt Dates 2011 and 2012, Dated: 08/06/2010
- RFA FD-09-001 for Receipt Dates 2009 and 2010, Dated: 12/18/2008
Past Press Releases