Discovery brings hope for muscular dystrophy sufferers
November 27, 1996
Web posted at: 10:30 p.m. EST
ATLANTA (CNN) -- Seven-year-old Chaz Payton has muscular dystrophy. If no treatments come along, he may only live to his early 20s.
"When he first was diagnosed, he was able to do stairs, he was able to get up off the floor," said his mother Shashana Payton. "Now he's weaker than that, he can't do stairs anymore."
Eventually Chaz's muscles will so weaken that he won't be able to walk and will have trouble breathing. The reason: he's missing an important muscle protein called dystrophen.
Scientists have tried -- unsuccessfully -- to find ways to replace the missing protein. There is currently no effective treatment for the disease, which affects about one in every 3,500 male births.
But Oxford University scientists say another muscle protein called utrophen may be able to fill in and do the work normally done by dystrophen.
"Utrophen can replace, functionally, dystrophen in muscle that's missing this protein, so that utrophen can rescue dystrophic muscle tissue," said Kevin Campbell for the University of Iowa College of Medicine.
To date, the utrophen experiment has only been done in mice. There's still a major hurdle before it can be tried in people: scientists must figure out how to get the extra utrophen into the muscles.
Scientists are not able to predict how long it may take for the utrophen discovery to become a viable treatment. But Dr. Brigitta Jann of the Emory Clinic said treatment with utrophen may one day slow down or even arrest muscular dystrophy.
"This particular discovery and its potential applications are closer to reality than many of the discoveries in the past," Jann said, while also cautioning against premature hope.
Chaz's mother said her family simply has to hope.
"We're very hopeful that there will be a cure in Chaz's lifetime ... that I'll be able to see my son actually run," Payton said.
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