Dr. Kunkel's laboratory discovered the gene causing Duchenne muscular dystrophy in 1986.
It can be used to check an embryo does not carry any one of 50 different conditions including cystic fibrosis and Duchenne muscular dystrophy.
Boys with Duchenne muscular dystrophy lack this protein, called dystrophin.
Jack Price, six, from Swansea, also has Duchenne muscular dystrophy.
Even with improved mechanical breathing assistance, people who have Duchenne muscular dystrophy the most common type of muscular dystrophy usually die of respiratory failure before they reach age 40.
Sarepta, formerly known as AVI Biopharma, boomed because of data from an 8-patient study that shows unprecedented efficacy for its drug, eteplirsen, dramatically reduced the rate at which patients with Duchenne muscular dystrophy worsened.
FORBES: Sarepta Outperformed Biotechs In August Due To Muscular Dystrophy Drug
Wyeth, one of the world's largest pharmaceutical companies, created a myostatin-blocking drug and put it into clinical trials for Duchenne muscular dystrophy, a muscle-wasting disease that kills hundreds of men each year before they reach their mid-thirties.
The sale will be preceded by a nine-city world tour beginning August 30 that will end with a three-day exhibition at the Monaco Yacht Show, which is co-organizing Only Watch with the Monaco Association against Duchenne Muscular Dystrophy and Antiquorum.
The benefits go far beyond the Duchenne muscular dystrophy, a disease that is diagnosed in only 600 American boys a year, to diseases like cancer and AIDS. Such drugs could even have a big effect on the muscle weakening that comes with aging.
One big question: would the limited data Sarepta has so far collected from a very small clinical trial of its drug eteplirsen, a treatment for Duchenne muscular dystrophy that is caused by a very specific type of mutation, be enough to warrant filing with the Food and Drug Administration and perhaps even gain approval?
FORBES: A Key Exchange On Sarepta Therapeutics' Promising Muscular Dystrophy Drug
Duchenne muscular dystrophy, discovered by French neurologist Guillaume Duchenne in 1868, accounts for 40% of the cases of muscular dystrophy in the U.S. In 1987 it became one of the first ailments linked to a defect in a particular gene, but since then the main treatments have been limited and unable to halt the disease's devastating effects.
Matt is one of 13, 000 boys in the U.S. who have a rare form of muscular dystrophy known as Duchenne.
In effect, Duchenne patients could develop a milder form of the disease, known as Becker muscular dystrophy, says Petra Kaufmann, a neuromuscular researcher who directs the Office of Clinical Research at the National Institute of Neurological Disorders and Stroke.
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