Posts By: coreygraham

Update: Focusing Forward

Dear Friends,   As we set out on the New Year, we’re looking back on what our community has accomplished and focusing forward on how to make more progress. 2016 was a monumental year, with the first-ever drug approval for Duchenne. We are proud to have been a part of that and grateful to everyone… Read more »

Breaking News: Eteplirsen was Approved

Today, the first safe and effective treatment for Duchenne muscular dystrophy—Eteplirsen—was approved. This is a proud day for every member of our Duchenne Community who worked for this moment and this historic victory—the experts who weighed in with the FDA, the community who rallied, and most of all the 12 boys in this trial who… Read more »

Share This 25-Second Video

We’re just three short weeks away from the critical FDA advisory committee meeting to evaluate eteplirsen on April 25th. This is a critical milestone for boys with Duchenne. Our community is coming together to ensure that the committee gets a full understanding of the science behind this safe and effective treatment. We need a roomful… Read more »

The Race to Yes: Where Do We Stand Today?

July 2012 Drug company Sarepta announces that after 36 weeks of dosing, DMD participants using eteplirsen outperformed the placebo. The company had demonstrated three months earlier that eteplirsen results in the production of significant levels of dystrophin, the protein boys with Duchenne are missing. December 2012 Sarepta requests a meeting with the FDA to determine… Read more »

The FDA’s Roller Coaster Ride for DMD Families

For months, the Duchenne community expected eteplirsen and drisapersen to be reviewed by an Advisory Committee on back-to-back days in November 2015. Now the FDA has delayed eteplirsen again until next year. This is unacceptable. The Race to Yes and patient advocacy organizations sent an urgent letter to the FDA demanding answers. As of November… Read more »

One Year Later: An Important Infographic Update

One year ago, you helped us rally more than 106,000 people to sign a White House petition urging approval for safe and effective treatments for Duchenne. Today, a full year later, we are still fighting for the fast and efficient regulatory approach that our children deserve. The FDA’s actions make it clear that they do… Read more »

Enough is Enough

Duchenne families to the FDA: Enough is enough. No more regulatory delay. It’s past time to say yes to safe and effective treatments for Duchenne Muscular Dystrophy The Problem Duchenne Muscular Dystrophy is an always fatal disease for which there is no currently available treatment. It affects about 1/3500 boys born every day. Boys are… 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 »