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Experimental gene therapy treatment for Duchenne muscular dystrophy offers hope for youngster
Date:2/7/2013

p>Functioning a bit like a bridge over a dangerous chasm, the experimental drug known as drisapersen is designed to effectively cover over the specific genetic mutation, allowing the problem area to be skipped and causing cells to produce a slightly shorter but functional dystrophin protein.

Because Duchenne muscular dystrophy is rare and the drug addresses only a small subset of the genetic variants responsible for the disease, recruiting qualified patients was not easy. Of the medical centers involved in the study, UC Davis, with its highly regarded neuromuscular disease and physical medicine and rehabilitation expertise, enrolled the largest group of patients in the nation. For more than a year, its eight young participants, including Jacob, have been to Sacramento from as far away as Colorado, Utah and Arizona. For each participant, the clinical trial involved weekly injections, which meant Jacob had to fly from Southern California to the UC Davis clinic every Friday for 24 weeks.

"I've never seen such a complicated study in terms of logistics," said Erica Goude, who serves as the research coordinator at the UC Davis site. "We're collaborating closely with departments of pediatrics, cardiology, radiology and several others, and their outstanding commitment to the project has made our tasks much easier and more efficient. This study is an amazing team effort that I see frequently reflected in the smiles of our patients and their families."

The study also entails extensive physical testing to monitor each participant's progress over time. To assess each child's physical abilities and progress, participants complete a six-minute walking test specifically designed and validated by a UC Davis team that included McDonald and Erik Henricson, a UC Davis muscular dystrophy researcher. The six-minute test is now used worldwide in all ambulatory clinical trials for Duchenne. Investigators also measure muscle strength and the level of dystrop
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Contact: Charles Casey
charles.casey@ucdmc.ucdavis.edu
916-734-9048
University of California - Davis Health System
Source:Eurekalert

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