After calling on the FDA directly to approve a breakthrough therapy for Duchenne Muscular Dystrophy, and appealing to Congress on behalf of the thousands of children facing this fatal genetic disease, concerned families have launched a national
"Last month we brought the world's leading scientists to the FDA and Congress to declare, 'This drug works,' said Tracy Seckler, whose son Charley faces Duchenne. "Last week, during Rare Disease Day parents went office to office in Congress with our urgent message for the FDA: our children need this safe and effective drug now. Now we're asking for over 100,000 signatures on the White House petition site by March 27th to get the President's attention regarding the urgent need for FDA approval."
"With a safe and effective treatment for our children awaiting FDA approval, we are in a sprint – the race to 'yes' - a race to get this and future therapies to our children," noted Marissa Penrod, whose son Joseph has Duchenne. "We believe that the FDA will see what the experts have already seen in this drug, and say 'yes.'"
About "The Race to Yes"
Duchenne is the leading genetic killer of children. The disease affects one out of every 3,500 boys born worldwide. With no approved treatment, Duchenne has a 100 percent fatality rate. A child of five diagnosed with Duchenne experiences a loss of muscle strength leading to wheelchair confinement by adolescence and a shortened life assisted by ventilators. The progressive deterioration of muscle strength leads to death in the late teens or early twenties. Today, leading scientists see a decisive decade for Duchenne research. The success of landmark clinical trials for the breakthrough drug eteplirsen is a pivotal moment that will lead to the development of additional new treatments for this dread disease. A pending FDA decision could determine whether this generation of children is the last to die from Duchenne, or the first generation to live. For more information including tools and resources concerning the "The Race to Yes" campaign, or to sign the White House petition, visit www.TheRacetoYes.org.
Media Contact: Norris Clark, Princeton Strategic Communications Group, 609-425-1325, firstname.lastname@example.org
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SOURCE The Race to Yes
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