Sunday, March 30, 2014

How eteplirsen works

Duchenne muscular dystrophy is caused by a mutation, or error, in the gene responsible for production of dystrophin, a protein that stabilizes muscle cells. Without dystrophin, normal activity causes excessive damage to muscles -- first limb muscles and later other muscles including the heart. Over time, the progressive damage causes muscle cells to die, and they are replaced by fibrotic tissue and fat.Chevron Paper StrawsEteplirsen works by directing the cellular machinery to skip exon 51, a small part of the dystrophin gene, to correct genetic mutations. That allows enough dystrophin production to transform Duchenne muscular dystrophy into Becker,Forklift forks a less severe form of the disease. About 13 percent of patients with the disease have genetic mutations that could be corrected with eteplirsen.
If approved, it would be the first drug on the market to treat muscular dystrophy.wholesale cheap Coach Grade AAA Sunglasses from homepage. Approval would encourage Sarepta and other pharmaceutical companies to develop more exon-skipping genes that address the larger number of Duchenne patients with other genetic mutations in the dystrophin gene."The longer it takes to get eteplirsen approved, the longer it will take to get started on [approving] these other compounds," including several that are nearly ready for clinical trials, Dr. Wilton said.That's a big concern for Amy Aikins of Seneca in Venango County.Our store can offer buy Dsquared Long Sleeve T-Shirts, welcome choose! Her 9-year-old son Elijah has a form of Duchenne muscular dystrophy characterized by a different genetic mutation. Eteplirsen won't help him, but another exon-skipping compound might.Dr. Mendell hopes approval of exon-skipping drugs like eteplirsen would lead to routine muscular-dystrophy tests for newborns so children can be treated before symptoms appear. Such tests already are available but are not done routinely because there are no approved treatments.honeycomb paper balls

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