New Drug May Reduce Symptoms Of Muscular Dystrophy

Laylah · Silver Member Joined: 27 Mar 2006 Posts: 178

New research found that a new therapy may be successful in replacing missing protein in patients diagnosed with Duchenne muscular dystrophy and by that slow the progress of this disease. This new experimental medication is an antisense fragment of RNA and it can reduce the effects of genetic mutation. People diagnosed with muscular dystrophy have a number of mutations in the gene that represents blueprint for dystrophin, a protein that plays significant role in muscle activity. Statistics show that people with this disease are bound to wheelchair by the age of 12 and most of them die by the age of 30.

This new drug works by binding to messenger RNA that’s produced from the defective gene and it enables body to produce more effective form of dystrophin. Scientists believe that this drug can reduce symptoms of muscular dystrophy .

During the research scientists investigated specific mutation in the dystrophin gene. Before treatment it was discovered that study subjects didn’t have dystrophin in leg muscles. After receiving injection of this experimental drug biopsy showed that 12% of tissue was composed of functional dystrophin. Patients didn’t have any side effects and this finding represents new possibility in treatment of this serious deadly condition. However, therapy is very expensive and more research needs to be done so it can be affirmed.

New Drug May Reduce Symptoms Of Muscular Dystrophy

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