CAMBRIDGE, Mass. — A drug used to treat Duchenne muscular dystrophy has been granted tentative approval and doctors are calling it groundbreaking.
For one local mother, this is the news she has been waiting to hear.
"I’m kind of speechless. It's, it's--there's been so many sacrifices and casualties along the way. So it's bittersweet," said Christine McSherry.
When Christine McSherry's son was just 5 years old, he was diagnosed with Duchenne muscular dystrophy. It effects one in about every 3,500 boys. Symptoms include frequent falls, trouble getting up or running, waddling gait, big calves and learning disabilities.
"It’s the most devastating diagnosis any mom can ever receive about their child, that they're going to have a short lifespan,” said McSherry.
Most patients don't live much longer than the age of 20 as muscle function deteriorates.
McSherry’s son is now 20. Two years ago, the family decided to partake in a small study for patients with Duchenne.
He was put on a drug called Eteplirsen, which is produced by Cambridge-based Sarepta Therapeutics. Within a few weeks, the McSherrys noticed a difference.
“He was able to open up a carton. He was able to open up a can. He snoring decreased,” McSherry told FOX25.
Advocacy groups wanted the drug made available for other patients right away, but that didn't happen.
Doctors at Mass General Hospital who worked on the drug believe the FDA was hesitant to approve it because the number of patients who had received it was small.
But on Monday, federal health regulators have granted temporary approval for doctors to administer the drug.
This will allow more patients to receive it, even as the full approval process moves forward.
McSherry said the long, slow process to move forward has cost lives, but she’s glad that more will now be saved.
“We can't forget the casualties that have happened. So while it seems like it might be a celebration, to more it’s more of a relief,” she told FOX25.
A doctor at MGH told FOX25 they're not sure what the timeline might be for full approval, but that this is a significant decision by the FDA to allow more access to the drug as the research into expands.
Cox Media Group
