Thursday, February 20, 2020

Thursday, February 20, 2020

FDA awards more than $4.1 million for natural history studies of rare diseases

The U.S. Food and Drug Administration (FDA) is providing more than $4.1 million in the next four years to fund two natural history studies of rare diseases.   

The research grant has been awarded to the University of Texas MD Anderson Cancer Center in Houston, and Vanderbilt University Medical Center in Nashville. The University of Texas MD Anderson Cancer Center’s research by Elizabeth Grubbs on the prospective study in medullary thyroid carcinoma will be receiving approximately $1.7 million over four years, while $2.4 million will be provided to the prospective study in cardiac disease in Duchenne muscular dystrophy by Jonathan Soslow of the Vanderbilt University Medical Center.

"The FDA is committed to funding these important studies in order to define how rare diseases develop and progress," FDA Principal Deputy Commissioner Amy Abernethy, M.D., Ph.D, said in a press release. "Further, these studies provide important roadmaps for how to conduct subsequent studies. With the natural history of many rare diseases remaining relatively unknown, efficiently developing diagnostics and therapies for patients poses unique challenges. Promoting and conducting work in this area is critical."

There were 31 grant applications that were received by the FDA and have been assessed for scientific and technical merit by more than 45 rare diseases, natural history, regulatory and statistical experts.

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U.S. Food and Drug Administration (FDA)

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