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MDA Awards $750,000 to Help Summit Test New Drug Formulation in Phase 1 Clinical Trial of Potential Treatment for Duchenne Muscular Dystrophy
Date:12/19/2011

y Price, Ph.D., Executive Chairman of Summit said:  "The new clinical study to be conducted on SMT C1100, a drug discovered and developed by our scientists is an important initiative.  We believe SMT C1100 has the potential to treat all DMD patients and provide benefit for them and their families."

A previous trial of an earlier SMT C1100 formulation on healthy volunteers by another biotech firm in California deemed the compound to be safe and well-tolerated.  However, variable blood concentrations were generated with only some individuals achieving expected therapeutic levels in the bloodstream.

About Utrophin Upregulation
Utrophin is a naturally-occurring protein that has a similar function to dystrophin.  Utrophin is produced during fetal development but its production is switched off in adults.  If utrophin production could be maintained, it could act as a substitute for the missing dystrophin to maintain the healthy function of muscles.  One method of turning utrophin production back on is through pharmacological means.  Utrophin upregulation has the potential to be beneficial to all DMD patients regardless of their specific genetic mutation and is also expected to be complementary to other therapeutic approaches in development.

About Muscular Dystrophy Association
MDA is the nonprofit agency dedicated to curing muscular dystrophy, ALS and related diseases by funding worldwide research.  The Association also provides comprehensive healthcare and support services, advocacy and education.

Long the world's leading non-governmental funder of muscular dystrophy research and health care services to affected individuals, MDA has played a pioneering role in utrophin-related research since 1996 (investing nearly $16 million to help develop this promising therapeutic pathway).   Presently, MDA active grants include 102 focused on Duchenne
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SOURCE Muscular Dystrophy Association
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