DMD research nears clinical trials

Researchers affiliated with Brown University and Tivorsan Pharmaceuticals Inc. in Providence are moving toward clinical trials of a treatment to combat Duchenne Muscular Dystrophy, the most common form of muscular dystrophy that kills males in young adulthood. More

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Focus: HEALTH CARE

DMD research nears clinical trials

PBN PHOTO/FRANK MULLIN
TEAMWORK: Brown University neuroscience professor Justin Fallon, second from right, with members of his research team, from left: Alison Amenta, Carolyn Schmiedel and Beth McKechnie. The team is searching for a treatment for Duchenne Muscular Dystrophy.
Posted 12/5/11

Researchers affiliated with Brown University and Tivorsan Pharmaceuticals Inc. in Providence are moving toward clinical trials of a treatment to combat Duchenne Muscular Dystrophy, the most common form of muscular dystrophy that kills males in young adulthood.

Justin Fallon, a professor of neuroscience at Brown since 1996 has been working 25 years on a possible cure for DMD and now, “we are entering the final stage of our development,” he told Providence Business News in a recent interview. “We’re encouraged with the progress we’ve made.”

“Encouraging developments are on the horizon,” predicted Dr. Joel B. Braunstein, acting CEO of Tivorsan Pharmaceuticals Inc., referring to what he called “intense interest” among researchers and drug companies across the country to identify an effective treatment for DMD with “a number of compounds” now being tested in various research arenas.

What Fallon and his seven-member research team are working on at Brown involves Biglycan, a human protein that can restore health to dystrophin-deprived muscles, reducing muscle degeneration and improving muscle function.

A recombinant, proprietary form of Biglycan has proven effective in treating mice with muscular dystrophy, Fallon said. Neither Fallon nor Braunstein could say when clinical trials on humans, which can last multiple years, might begin. “We are in preclinical development,” Braunstein said. “We are working to advance this program as quickly as possible.”

Duchenne Muscular Dystrophy is an inherited condition, caused by a mutation of genes that robs muscles of dystrophin. It affects approximately one in 3,500 newborn boys and is usually diagnosed at the age of 4 or 5 years old.

For a few years after diagnosis, the afflicted boys are “OK, they are really fine,” Fallon said.

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