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SMT C1100 For Duchenne Muscular Dystrophy Moves To Human Testing
Date:4/24/2012

TUCSON, Ariz., April 24, 2012 /PRNewswire-USNewswire/ -- The Muscular Dystrophy Association announced today that SMT C1100, an experimental drug for Duchenne muscular dystrophy (DMD), has received approval from regulatory agencies in the United Kingdom to move from laboratory to human testing, starting with healthy human volunteers.

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The drug is designed to increase production of a muscle protein utrophin that can potentially compensate for the one that's missing in DMD.

The Association has been supporting the development of SMT C1100 through its MDA Venture Philanthropy drug development program, via a grant to Summit Corporation PLC of Oxford, United Kingdom.

"We're extremely pleased that SMT C1100 will now move from testing in vivo to testing in healthy humans," said Jane Larkindale, MDA's director of translational research. "We have good evidence from laboratory studies that this drug's mechanism of action is valid for slowing the progression of this degenerative muscle disease, and we hope that this first trial will show that it can be effectively and safely delivered to humans."

Summit's Chief Scientific Officer Richard Storer said: "Utrophin upregulation will be beneficial to all DMD boys, and SMT C1100 has demonstrated disease-modifying potential in nonclinical efficacy studies. This trial will evaluate if our improved formulation of SMT C1100 can produce consistent levels of the drug in the blood which, based on the preclinical studies, might be expected to have a therapeutic benefit in patients."

About SMT C1100

SMT C1100 is designed to boost production of the muscle protein utrophin. Utrophin closely resembles and can mimic some of the functions
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SOURCE Muscular Dystrophy Association
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