With tantalizing early results, Sarepta’s gene therapy for Duchenne raises hopes for ‘real change’
An experimental gene therapy for Duchenne muscular dystrophy, licensed to Sarepta Therapeutics, produced jaw-dropping increases in a crucial muscle protein normally missing in patients with the disease, according to preliminary clinical trial data released Tuesday.
The data were collected from just three boys, but the effect of the gene therapy — producing 38 percent of a truncated form of the normal dystrophin muscle protein — is profound enough to suggest it may halt or even reverse the effects of Duchenne in certain patients.
“I have spent my life wanting to make a real change in this disease. Finally, we may be there. I am very hopeful. This is an emotional time for people in the field,” said Dr. Jerry Mendell of Nationwide Children’s Hospital in Columbus, Ohio, speaking to STAT on Monday
