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Experimental Treatment Could Fight Muscular Dystrophy

Posted: October 18, 2010 at 8:33 am


(HealthDay News) -- Injecting a therapeutic molecule into muscle appears to jump-start the production of a crucial protein that's missing in patients with Duchenne muscular dystrophy, British researchers report.

The treatment so far is only applicable to about 13 percent of people with the debilitating and ultimately fatal disease, but scientists are hopeful that similar molecules might expand the treatment to a wider range of patients.

Duchenne muscular dystrophy affects about one in 3,500 males, and involves a progressive wasting of muscle due to a genetic inability to produce the protein dystrophin, a key component of muscle structure. No treatments are available for the illness, and most of those affected die by age 30.

Recently, molecules called antisense oligonucleotides have shown some promise. These molecules work by "skipping over" portions of the defective gene that would otherwise block dystrophin production. Read more...

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