Monday, March 31, 2014

I desperately wanted drisapersen to work

"I desperately wanted drisapersen to work. The bad news is that [the drisapersen trial results] cast a specter over the entire field," Dr. Wilton said. "To me, this is the same as saying one early-generation antibiotic did not clear up a bacterial infection,Atlanta Falcons Jason Snelling iPod Touch 5 CaseCustom LOL lovely Q version design pure cotton long sleeve T-shirt hence all antibiotics do not work."Dr. Mendell said the FDA seems to want data that doesn't exist yet."I don't think we know in Duchenne dystrophy whether replacing dystrophin alone would be adequate to improve function.Custom Rammus LOL awesome hatPure Cotton Long Sleeve And we just don't know how much dystrophin we need to improve clinical function. There are no measures for that because it's never been done before now,Honeycomb Tissue Balls" he said.Sarepta is prepared to conduct additional trials of its drug. Chris Garabedian, Sarepta's president and CEO, was hoping the FDA would give preliminary approval to take the drug to market even as trials are conducted to confirm eteplirsen's effectiveness, but that appears less likely now.
Still, he is continuing to press for fast action."We continue to believe that the currently available data for eteplirsen could be sufficient to qualify for consideration of an early approval with the understanding that we'll conduct a larger study to confirm the findings," he said. "We are working with the FDA to address their concerns and get their agreement that an application for eteplirsen is worthy of a formal review."Mr. Garabedian is confident that approval will come eventually. The key is whether it happens soon enough for the thousands of Duchenne boys whose muscles are rapidly weakening.Parents know that side effects could emerge years down the road, but that's a risk they are willing to take to save their dying boys."Parents are willing to take the risk because we have no alternative," Mrs. Ellsworth said. "The alternative is death."

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