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Fragile X

Rare disease collaboration receives NIH funding for a new Center for Fragile X disease

10/22/2020

An interview with Peter Todd, M.D, Ph.D., Neurology, Medical School, University of Michigan (U-M), and member of the Executive Committee of the U-M Center for RNA Biomedicine

Dr. Todd will serve as co-director of a Fragile X Center without walls with investigators at U-M, Baylor College of Medicine, and Emory University. This Fragile X Center is one of three recently funded by National Institutes of Health (NIH). Fragile X syndrome is the most common inherited form of intellectual and developmental disability (IDD), and the repeat that causes Fragile X Syndrome can also result in neurodegeneration and premature ovarian failure. The center will receive $9 million over the next 5 years.

“Rare diseases” are not rare
First off, rare diseases are not rare in aggregate- one in 10 (~30 Million) people in the US will suffer from a rare disease. Second, in many of these rare diseases, we have been able to define their genetic basis, and that has allowed us to model them effectively. This has led to targeted and effective therapies for a number of previously untreatable rare conditions. [Read more…] about Rare disease collaboration receives NIH funding for a new Center for Fragile X disease

Filed Under: News Tagged With: Fragile X, Peter Todd M.D Ph.D, rare disease, RNA sequence repeats

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