BOSTON (CBS) – A medical conference in San Francisco is still buzzing in reaction to some positive clinical results from a Cambridge biotech which is trying to attack a disease that kills children.
Sarepta Therapeutics says its first ever investigational drug for Duchenne Muscular Dystrophy is showing real promise.
Sarepta CEO Chris Garabedian tells me it’s one of the most severe forms of Muscular Dystrophy.
“It’s 100% fatal. It affects mostly boys, and they usually pass away in their late teens and early 20s,” Garabedian says.
He says he and his team have been working for years to find a way to stop or slow the progression of Duchenne.
“We have over two years of data that show we’ve generally stabilized the walking ability of these patients in our study,” he adds.
Garabedian also thinks the technology could be transferable to other diseases.
“We just talked about how we can modulate protein expression and gene translation across a lot of different applications in the neuromuscular diseases, other rare diseases, and we think this technology could even help with oncology or immune inflammatory diseases.”