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Sarepta Therapeutics, Inc. Message Board

  • zwerp2000 zwerp2000 Oct 30, 2013 2:12 PM Flag

    Jett Foundation

    Posted by Christine on October 30, 2013

    Katie (Katherine) at the Jett Foundation was thrilled to receive this email today from Elizabeth Warren!! Momentum is building. This is another great step in our push for Accelerated Approval


    Dear Katherine of Jett Foundation,

    Thank you for contacting me to express your support for the accelerated approval of the drug eteplirsen, which has the potential to help a subset of boys with Duchenne Muscular Dystrophy (DMD), by the FDA.

    DMD is a fatal genetic neurological disorder and is one of the several types of muscular dystrophy. According to the Center for Disease Prevention (CDC), it affects approximately one in 3,500 boys born in the country. More than 20,000 boys and young men are estimated to suffer from DMD. The disease is caused by a mutation in the gene that produces the protein dystrophin. Eteplirsen was made possible due to our understanding of the genetics of DMD, and I believe that our investments in research will continue to give us new targets and therapies for DMD.

    I appreciate your views on this issue and recently sent a letter with my colleague Senator Ed Markey urging the FDA to consider the application for this drug. In addition, I strongly support increasing our government’s investment in scientific research to build the knowledge base necessary to advance medicine. For that reason, I supported an increase in funding during the appropriations process for the National Institutes of Health (NIH), our nation’s premier medical research agency and funder of academic medical research around the country. I will continue to fight alongside my Senate colleagues to provide research funding that will help lead to better insights and new treatments for those with DMD.

    I appreciate your reaching out to me about this important issue, and I hope you contact my office again in the future about issues of importance to you or if I can be of assistance.


    Elizabeth Warren
    United States Senator

    Dear Senator Warren,
    Thank you so much for your response. We are thrilled by your show of support and grateful for your help. In particular, thank you for signing a letter with Senator Ed Markey to urge the FDA for Accelerated Approval for eteplirsen. We are fighting to save lives and this drug is need by boys with Duchenne immediately. We need help getting the support of your colleagues in Congress. Would you be willing to help champion this cause and get others to support our efforts?
    Thank you for your help and your consideration. I look forward to hearing back from you.
    With best regards,
    Katie Cutler
    Development Officer

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