New Drug Offers Hope Of Treating Duchenne Muscular Dystrophy
(CBS) -- It's the most common fatal childhood disease: Duchenne muscular dystrophy. Now results from a new drug trial are offering families hope, reports CBS 2's Roseanne Tellez.
Just walking is a challenge for 13-year-old Nicholas Gonzalez who has Duchenne muscular dystrophy. The progressive muscle wasting disease that puts most boys his age in a wheelchair full-time.
Mom Michelle jumped at the chance to put Nicholas in a clinical trial of a drug called Drisapersen. After one year, she says the muscle deterioration has slowed dramatically.
"As part of a puzzle, this is something that has shown wondrous effects," Michelle said. "I've seen children walk, run, ride bikes."
"I've been able to walk a little longer," Nick said. "Been able to be more independent."
But without FDA approval, boys like Nick will have no treatments to turn to. That's why Gonzalez, and other parents like her recently testified before an FDA advisory committee.
"The FDA, life or death, it's in their hands," she said.
Debra Miller, with Cure Duchenne talked to us via Skype.
"The boys want this drug, their parents want this drug, and what I think is even more important the physicians that are treating these boys want this drug," Miller said. "We hope that one day this will be a manageable disease and that it will be where when your son is diagnosed with Duchenne, it's not a death sentence."
One mom started a petition on Change.org that got more than 100,000 signatures in 10 days.
Roseanne's son David has Duchenne, but this drug would not benefit him, because it targets only a certain subset of boys with Duchenne. But the hope is, if approved, the pharmaceutical companies would then target other subsets, so this is a really hopeful time.
