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New clinical trial results show how personalized medicine will alter treatment of genetic disorders
Date:12/26/2007

reactivate dystrophin protein production in small areas of muscle tissue at the injection site of muscular dystrophy patients.

"Dozens of specific sequences will be required for effectively treating the majority of patients with Duchenne muscular dystrophy," writes Dr. Hoffman. "But in order to realize the promise of personalized molecular medicine in muscular dystrophies and, ultimately, other disorders, it will be important to re-evaluate current measures of toxicity, efficacy, and marketing that ensure both safety for the patient, as well as rapid development and distribution of life-saving drugs."

Dr. Hoffman envisions that some parts of the approval process may be developed for DNA-like molecular medicine as a 'class' of drugs, rather than individual testing of hundreds of different sequences.

"The patients and their families are crossing their fingers that the drugs overall chemistry can be shown to be safe," he says.


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Contact: Jennifer Leischer
jleische@cnmc.org
202-476-4500
Children's National Medical Center
Source:Eurekalert

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