FDA: Please, approve the medicine my boys need to survive -- both of my sons deserve to live
  • Petitioned Dr Russell Katz

This petition was delivered to:

Food and Drug Administration
Dr Russell Katz
FDA
Dr Anne Pariser

FDA: Please, approve the medicine my boys need to survive -- both of my sons deserve to live

    1. jenn mcnary
    2. Petition by

      jenn mcnary

      saxtons river, VT

My two oldest children, Max and Austin, have a lethal genetic disease called Duchenne Muscular Dystrophy (DMD). Right now, only one of my sons has access to a new "miracle drug,” and it has changed our lives. I am watching one of my boys thrive, as my other gets tragically worse while he waits for access to the medication that could save him.



Every day, children with DMD get weaker, it causes loss of muscle, to the point where children with the disease cannot breathe on their own. It is a slow death sentence.



Miraculously, one of my sons has been saved. Max, just 10 years old, was lucky enough to take part in a medical trial. For the last 60 weeks, Max has been taking this new medication. This year, my son who was once dependent on a wheelchair most of the day, marched in a Halloween parade.



But our journey has been bittersweet. As we watch Max get better, we also watch his older brother, Austin, 13, get worse. He suffers, silently, as his disease progresses. Austin was unable to take part in the drug trial that helped his brother so much.



Although Austin doesn't walk, he still has strength in his upper body, which this drug could help him keep. It could save him from spinal surgery, a tracheostomy, a feeding tube. It could allow him to drink and eat. It could keep him sitting up. It could allow him to hug his family.



Eteplirsen is the first hope we have had since Austin and Max were diagnosed with Duchenne. It has the ability to stop this disease in its tracks...and the boys who need this drug simply don't have one more day to waste, waiting for it. Austin does not have one more day to waste, waiting for it.



This development comes at a very important time. A law was just passed, allowing the FDA to move drugs like these, for fatal illnesses, like Duchenne, through an accelerated approval process.

Thousands of boys in the United States are suffering with DMD. If the FDA allows American boys access to this drug, it will save thousands of lives -- and could help many more across the globe get the care they need.

Please, Austin and Max both deserve a chance to thrive. Tell the FDA to make this important drug available to all children with Duchenne Muscular Dystrophy.


Recent signatures

    News

    1. Reached 150,000 signatures
    2. We are gaining momentum and support

      jenn mcnary
      Petition Organizer
      Mom urges drug approval to help her son : Rutland Herald Online

      Rutland Herald Online - Vermont election news, news coverage, local news, state government, sports, classifieds

    3. Reached 12,500 signatures
    4. Week 62 data on Eteplirsen

      jenn mcnary
      Petition Organizer

      We are excited to see continued stability on the 6-minute walk test with more than a year of follow-up where we would otherwise predict a significant decline," said Chris Garabedian, President and CEO of Sarepta Therapeutics. "Furthermore, the placebo / delayed treatment group has shown stability over the last 26 weeks of treatment, the period in which we would expect to see dystrophin levels translate to clinical benefit. These clinical data support our belief that the dystrophin levels we observed at 48 weeks are potentially an important surrogate marker in assessing disease progression in Duchenne and renews our commitment to advance this program forward as we prepare our primary 48 week dataset for discussion with the FDA." http://investorrelations.sareptatherapeutics.com/phoenix.zhtml?c=64231&p=irol-newsArticle&ID=1765390&highlight

    5. Reached 10,000 signatures

    Supporters

    @ParentProjectMD

    Nov 29, 2012

    Recent petition to the FDA by #Duchenne mom @JennMcnary is gaining traction... Over 6,000 signatures already! http://t.co/DOUlzJWa

    Parent Project MD
    1.28k followers

    @ActionTilson

    Dec 08, 2012

    FDA: Please, approve the medicines for #Duchenne “I don’t want to die the thought of that frightens me.” http://t.co/jAszdvxB

    Carl ActionTilson
    1.12k followers

    @angelayee

    Dec 06, 2012

    Sign this petition so the FDA can accelerate approval on a miracle drug to treat Muscular Dystrophy, don't waste time! http://t.co/9OX9K0R2

    Angela Yee
    249k followers

    @ryanlochte

    Nov 29, 2012

    Help me & my pals @ParentProjectMD support @jennmcnary. Sign the petition & tell the FDA to #EndDuchenne! http://t.co/kWENJumw #jeah

    Ryan Lochte
    1.01M followers

    Reasons for signing

    • Maryellen Brady EDGEWATER, AL
      • 2 months ago

      Kids matter and we the people should be taking care of all our kids.

      REPORT THIS COMMENT:
    • Royce Pellegrin HOUMA, LA
      • 2 months ago

      I Want To Help.

      REPORT THIS COMMENT:
    • Melissa VanderLely ST CATHARINES, CANADA
      • 2 months ago

      My son has DMD and we are desperate for something, anything that will help him.

      REPORT THIS COMMENT:
    • Barbara Daniels VINELAND, NJ
      • 3 months ago

      Let it not be about the money!!!

      REPORT THIS COMMENT:
    • James Himmel GLENVIEW, IL
      • 3 months ago

      My son died 2 years ago. Too late to help him, but we need to save the rest with this drug

      REPORT THIS COMMENT:

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