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Brown licenses potential muscular dystrophy treatment to Tivorsan Pharmaceuticals

w treatment in DMD. If biglycan proves safe and effective in human testing, the potential exists for providing relief to thousands of boys and their families around the world."

Fallon serves as chair of Tivorsan's scientific advisory boaard.

Developing biglycan

In 2000 a team led by Fallon began to accumulate evidence that biglycan, which is produced naturally in the body, regulates the production of a protein called utrophin. Utrophin, discovered in 1989, stimulates the body to repair muscle damage, just like dystrophin, the protein that DMD-afflicted boys are genetically unable to produce.

All young children produce utrophin, but they produce much less of it naturally as they get older.

"In normal individuals that's fine because dystrophin is there, but in a Duchenne boy there is no hand-off," Fallon said. "The baton is dropped."

That dropped hand-off helps explain why DMD doesn't begin to show up in boys until they are preschoolers and why they still retain enough strength to walk on their own until they are preteens.

Further research by Fallon and research associate Beth McKechnie revealed that biglycan is rare in its class of proteins because the physiologically active form can be manufactured relatively easily using recombinant lab techniques. In the last several years, Fallon's research group, including former research associate Mary Lynn Mercado and current research associates Alison Amenta and Michelle Dechene, have been testing and refining the use of biglycan in DMD mice.

Partners prepared to fight

The research has gone well enough that last year Fallon won a four-year, $5.2-million grant from the National Institutes of Health to advance the development of biglycan as a novel potential therapy.

Fallon also won funding from foundations dedicated to fighting DMD. Last year Parent Project Muscular Dystrophy awarded him its End Duchenne Award. Charley's Fun

Contact: David Orenstein
Brown University

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