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Spring 2014
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Doing It for the Kids
A brain scientist takes a detour into muscle.
Justin Fallon and his team of researchers work in the dynamic spaces between one reality and the next—the placeswhere synapses fire to catalyze creative thought and long-term memory, andwhere basic science may one day morph into a clinical therapy for children suffering the cruel fate of muscular dystrophy.

Fallon was recently awarded a $5.3 million grant from the National Institutes of Health (NIH) to continue to build  on research suggesting that Duchenne muscular dystrophy—the most common form of the disease—may be treated with a new therapy. The new approach would be designed to prompt wasting muscle cells to express a specific fetal protein, replacing another essential protein no longer produced by those afflicted with the disease. The grant is milestone-driven, delivering more than $1.3 million annually to the project for four years as long as progress is being made. The earlier research was funded by the NIH along with three advocacy groups—Charley’s Fund, Parent Project, Muscular Dystrophy (PPMD), and the Nash Avery Foundation.

Pockets of hope
Duchenne muscular dystrophy rides stealthily in on the X chromosome. It is a hereditary disease caused by a mutation in the gene that contains instructions for making a protein called dystrophin, which strengthens muscle fibers and protects them from injury as muscles contract and relax. Duchenne wastes and weakens little boys’ muscles as toddlers, puts them in wheelchairs by puberty, and takes their lives in young
adulthood.

There is no treatment. There is no cure. There are just pockets of hope.

“In the absence of effective treatment, it’s very important for parents to know at least that people are working on the disease,” says Fallon. “And we are. There is a significant aggregate effort [of research targeting Duchenne]. The more shots on goal, the better. The big question is Which one is going to work, when?”


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