Sarepta's announcement on the continued show of progress and promise in their product eterplisen is great news and a great way to begin the new year.Week 120 and counting.Considering it hasn't exhibited any adverse safety concerns so far,it gives one still more hope to face another day for our sons.

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Sarepta Therapeutics Inc at JPMorgan Healthcare Conference

its realy a great news, i hope that fda makes this drug passed for our dying boys as soon as possible, we dont have enough time to waste!

WE need PPMD and all of the advocacy groups to unite to put massive pressure on the FDA to get this drug approved.

This is an article about an MS drug that is having a similar problem with the FDA. We need to unite with all rare disease groups and fight the FDA's stupid adherence to protocol regardless of circumstances.


The Wallstreet Journal

The FDA Nixes a Pathbreaking Drug for MS

Thirty developed nations have approved Lemtrada. The U.S. refusal to do so shows the need for regulatory reform.

Makes one wonder why Sarepta doesn't just give up and restart the trial in Canada
I mean it's not like Americans aren't used to ordering drugs from Canada. My US doc writes a prescription for online pharmacy and poof! 5 days later I have meds. Why would this be different? I suspect this is easier than changing any party of American government. SAREPTA GO NORTH!

Hello David.I think Sarepta should follow your line of thought on this one very closely.I mean really,why not move to Canada if that is where the key to rapid approval may lie?Of course we all would love to understand FDA's drugs safety and effectiveness concerns but from where they stand they must surely have a very clear view of the desperation that our families and sons are living in and the fact that time is not for us.Really if we are putting our sons on steroids despite all the known adverse side effects and their less than spectacular results in halting DMD,it is only fair they allow us access Sarepta's product whose effects are all positive so far minus any known adverse side effects.Sad.  

The same is about Ataulren which could have been approved a few year ago basing on IIb trial

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