Important Update: 168 Week Data Released

Dear Friends: This week, Sarepta Therapeutics released 168 week data from its ongoing eteplirsen study.  This new data strongly confirms the safety and efficacy of eteplirsen and once again calls into question the unreasonable delays and barriers to approval raised by the Division of Neurology Products at the FDA. According to a company press release,… Read more »

Eteplirsen Works on Legs and Lungs…If It Gets to them Fast Enough

By Tracy Seckler Sarepta’s 168 Week Data Affirm Safety and Efficacy Early this morning, Sarepta Therapeutics released more data from the company’s long-term clinical trial testing eteplirsen. With the release of this data, I am heartbroken. But not because the results disappoint. I am crushed because this treatment is so promising, so safe, and so… Read more »

Watch Our Google Hangout Here

In recent days, a series of difficult developments once again put the path to approval just out of reach. We need you to join the next lap of this increasingly challenging course. We need you to know first hand exactly what is happening, that’s why we laid it all out in this Google Hangout.

Important: Google Hangout This Thursday, Noon EST

Dear Race to Yes Supporter, It’s been an extraordinary few months since the White House answered over 106,000 Americans who joined the Race to Yes. Because of you, decision makers in Congress and the FDA began to accelerate the process of making safe, effective treatments available to childrenwith Duchenne. You changed the world. Regrettably, the… Read more »

Act Now: 2 Ways You Can Help the Race to Yes

Thank you for all your efforts in The Race to Yes! We wanted to give you an update and ask for your continued help to win this Race. The Facts Earlier this month, natural history data was released that underscores how absurd it is to suggest that the stability of the boys with Duchenne receiving… Read more »

Race to Yes Responds to White House

Washington, DC, July 30, 2014 – In response to the 106,782 signatures collected through TheRacetoYes.Org petition urging the “FDA to Say Yes to Accelerated Approval for safe, effective therapies for children with Duchenne” muscular dystrophy, the White House issued a statement yesterday by Janet Woodcock, Director, FDA Center for Drug Evaluation and Research. The Race… Read more »

White House Responds, but Time is Muscle

As many of you know, after gathering more than 106,000 signatures and then waiting over four months, last night we received a response to our White House petition asking for accelerated approval for safe, effective treatments for Duchenne. We are glad that at last, the White House is publicly acknowledging innovative pathways are needed and… Read more »