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Muscular Dystrophy Association Awards $13.7 Million for 40 New Research Grants
Date:8/23/2011

is exploring a new way to up-regulate utrophin, a muscle protein that may be able to stand in for the larger dystrophin protein that when absent causes DMD.  Plus, an MDA-sponsored team in Victoria, Australia is working to prevent extensive bone loss caused by corticosteroids, the only available treatment shown to slow the progression of DMD.

For ALS, a Houston-based team with MDA funding is investigating whether a combination drug treatment (Licofelone + Riluzole) works better than the first FDA-approved drug for ALS, riluzole.  Another group of MDA-sponsored investigators in Quebec City, Canada is looking for the earliest visible signs of ALS.  Finally, a San Diego team supported by MDA is using next-generation gene sequencing technology to better understand what makes ALS-causing mutations in two genes (TDP-43 + FUS/TUS) so crucial to the ALS disease process.

With tens of millions of Americans affected by type 2 diabetes and 30 percent of FA patients developing diabetes, a new MDA-funded project in Philadelphia to determine the exact mechanisms by which diabetes occurs in FA also could shed valuable insights into the causes of insulin resistance seen in people with type 2 diabetes.  

Other exciting initiatives by MDA-funded investigators include the Minneapolis-based effort to identify inhibitors of the DUX4 gene implicated in FSHD, and to quickly test promising compounds in a transgenic mouse model for that disease; and, a Miami-based effort
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SOURCE Muscular Dystrophy Association
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4. PTC Therapeutics and Parent Project Muscular Dystrophy Advance Drug Discovery Program to Improve Heart Function in Duchenne/Becker Muscular Dystrophy Patients
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