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PrintMedical Reports: Muscular Dystrophy
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Leisa Williams
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Muscular dystrophy is one of the most common genetic disorders affecting children. The most devastating type slowly robs kids of their muscles, and they rarely live past age 20. But now, researchers say they've made a drug discovery that could give kids a fighting chance. 7's Christine Cruz has the details in today's Healthcast.
WSVN -- He's gradually getting weaker, but his star power is stronger than ever.
Drew Bonner is an inspiration to kids with Duchenne Muscular Dystrophy, a fatal genetic disorder where muscles waste away.
Jan Bonner: "I said, 'OK, what are the treatments, where do we go from here? We can deal with this,' and the doctor told us, 'There are no treatments, there are no cures.'"
He doesn't depend on a wheelchair yet but does lean on others to get around.
Drew Bonner: "It's a challenge I have to overcome. It's one more hurdle to jump over."
Researchers say they've cleared a huge hurdle in finding a treatment.
Dr. Eric Hoffman, Children's National Medical Center: "It's at our fingertips, getting these kids out of their wheelchairs."
Dr. Eric Hoffman is part of a world-wide team developing the technique called Exon-skipping. The injectable drug cocktail works like a band-aid, covering up the mutation that causes muscle weakness, allowing cells to make a healthier protein that improves muscle function.
Dr. Eric Hoffman: "It's almost as if you were reading a recipe and instead of putting salt, your mutation said put in lots of hot pepper instead. Well, your cake wouldn't be so great. This band-aid lets it skip over that wrong pepper instruction."
When tested in dogs with muscular dystrophy, the animals went from struggling to walk to running, which is a good sign since the canine version of the disease affects the same gene as the human version.
Jan Bonner: "Time is not on our side."
So far, the drug cocktail is only being tested on people in Europe. Drew says for him, he'll keep living his life while hoping for a cure.
Drew Bonner: "How's life any fun if you're just gonna be a downer? Why not just live it and be happy."
Researchers say this strategy could help up to 90 percent of people affected by Duchenne Muscular Dystrophy. There's no indication when testing will start here in the U.S.
FOR MORE INFORMATION, PLEASE CONTACT:
Genetic Counseling
Children's National Medical Center
(202) 476-4925
http://www.childrensnational.org/