The Race to Yes Heats Up!

Posted by: Christine McSherry on March 13, 2014



The Race to Yes is a movement to expedite approvals for new medicines that are safe and effective for people with Duchenne.  Today, the Race to Yes is about Accelerated Approval for eteplirsen because it is the only drug that is a candidate for Accelerate Approval at this time.  The past week has proven that this campaign is truly expanding.  We have gotten 25,000 signatures on the White House petition, and many of those have come from widespread support of various Duchenne organizations.  The community (Thank YOU Parent Project Muscular Dystrophy and The Muscular Dystrophy Association!) has come together to advocate for approval of the first safe and effective treatment for Duchenne.  And we will stay together to advocate for any and all safe and effective treatments, because we know that more are coming.  As Harvard’s Dr. Louis Kunkel told Congress, “This is the decade of therapy.”

Our community lives with Duchenne every single day.  We understand that an additional three to five years of waiting for approval of a drug that is safe and effective is unacceptable.  This first drug is a life-line for some.  For all of us affected by Duchenne and rare disease, it blazes a new pathway for follow-on exon skipping compounds and other therapeutic approaches to get approved.


Thanks to all who have joined the Race to Yes by promoting the White House petition.

Now on to One Hundred Thousand!!


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