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Home > MDA Awards $750,000 to Help Summit Test New Drug Formulation in Phase 1 Clinical Trial of Potential Treatment for Duchenne Muscular Dystrophy

MDA Awards $750,000 to Help Summit Test New Drug Formulation in Phase 1 Clinical Trial of Potential Treatment for Duchenne Muscular Dystrophy

TUCSON, Ariz., Dec. 19, 2011 /PRNewswire-USNewswire/ -- The Muscular Dystrophy Association (MDA [1]) has awarded $750,000 to Summit PLC [2], a UK biotechnology company, to continue development of an experimental drug that may become a viable treatment for boys with Duchenne muscular dystrophy (DMD [3]).

The funding comes from MDA Venture Philanthropy (MVP), a part of MDA's translational research program.

DMD is one of nine types of muscular dystrophy, a group of genetic, degenerative diseases primarily affecting voluntary muscles.  Caused by mutations in the gene that makes dystrophin, a protein that normally protects muscle cells and keeps them intact, DMD eventually affects all voluntary muscles, and the heart and breathing muscles.  DMD affects 1 in 3,500 boys with an estimated patient population of 50,000 in the developed world.    

Summit's SMT C1100 is an experimental compound designed to increase the muscle protein utrophin, which is a naturally-occurring protein that has a similar function to dystrophin.  Investigators are confident that if utrophin could be upregulated sufficiently, it could act as a substitute for the missing dystrophin in DMD to help maintain healthy muscles.

"A long-time champion for utrophin upregulation as a promising therapeutic strategy for treating Duchenne muscular dystrophy, MDA is pleased Summit soon will be testing a new formulation of SMT C1100 in a human clinical trial," said Valerie Cwik, M.D. Executive Vice President Research and Medical Director for the Muscular Dystrophy Association. 

"This is a vital step toward knowing definitively whether utrophin can successfully stand-in for the missing dystrophin protein that MDA-funded investigators identified as the underlying cause for this form of muscular dystrophy," Cwik added.

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Summary: 
TUCSON, Ariz., Dec. 19, 2011 /PRNewswire-USNewswire/ -- The Muscular Dystrophy Association ( MDA ) has awarded $750,000 to Summit PLC , a UK biotechnology company, to continue development of an experimental drug that may become a viable treatment for boys with Duchenne muscular...
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Source URL: https://www.mdtmag.com/news/2011/12/mda-awards-750000-help-summit-test-new-drug-formulation-phase-1-clinical-trial-potential-treatment-duchenne-muscular-dystrophy

Links
[1] http://www.mda.org/
[2] http://www.summitplc.com/
[3] http://www.mda.org/disease/dmd.html
[4] http://www.bio-medicine.org/medicine-technology-1/MDA-Awards--24750-000-to-Help-Summit-Test-New-Drug-Formulation-in-Phase-1-Clinical-Trial-of-Potential-Treatment-for-Duchenne-Muscular-Dystrophy-23030-1/
[5] https://www.mdtmag.com/content-item-types/news