Biopharmaceutical Company Encouraged by Duchenne’s Clinical Trial

AVI BioPharma, a biopharmaceutical company based in Corvallis, Ore., said a recent clinical trial to treat Duchenne’s Muscular Dystrophy was successful.
The trial involved the use of a potential drug developed by AVI BioPharma called AVI-4658. Boys who have Duchenne’s and were participating in the trial were injected in one foot with AVI-4658, and with a saline placebo in the corresponding muscle of the other foot.

Several weeks later, AVI BioPharma reported, the injected muscles were biopsied and examined to see if dystrophin production had been stimulated. Dystrophin is a protein critical for muscle fiber function, and patients with Duchenne’s Muscular Dystrophy “have a very low capacity to make dystrophin,” AVI BioPharma said.

The company said the AVI-4658 injections “elicited dystrophin production in a dose-dependent manner in all treated patients” and added that the drug was “well tolerated, with no significant, detectable drug-related adverse effects.”
The study was conducted in the United Kingdom by members of the MDEX Consortium, described as a “multidisciplinary enterprise to promote translational research into muscular dystrophies.”

Improving dystrophin production in patients, AVI BioPharma said, could significantly slow the disease’s progress and improve the quality of life for boys and young men with Duchenne’s Muscular Dystrophy. For more information on the clinical trial, visit www.avibio.com.

Duchenne’s is a genetic condition that causes progressive muscle weakness and loss, leading to compromised mobility, respiratory distress and eventual paralysis. There is currently no cure, and treatments center around physical therapies to improve muscle strength and quality of life and independence. The National Library of Medicine estimates about 1 in 3,600 boys is born with Duchenne’s Muscular Dystrophy.

This article originally appeared in the Seating & Positioning Handbook: March 2009 issue of Mobility Management.

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