Additional urgency for accelerated approval of eteplirsen from parents.
Posted by: Christine McSherry on September 24, 2013
As one of parents who have been advocating for a faster pathway of eteplirsen – there are several major points that we feel need to be communicated to the Duchenne community and the FDA. I believe that there could be data pulled from drisapersen study that could strengthen and enhance the argument for accelerated approval for eteplirsen.
The outcome and natural history of Duchenne is known. The outcome is always the same. Those diagnosed who start to decline – don’t get better, ever. By not doing anything – by delaying a potential therapy to those who are sentenced to death, is worse than doing something. The parents have told the FDA that they are willing to take permissible risk. The FDA has all the information it needs to make an informed decision. The data stands on its own, as do 10 of the boys in the trial — and 2 young guys who still have tremendous upper body strength – if there were ever a time to be lucky when diagnosed with Duchenne – being included in the eteplirsen trial is lucky.
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