The Race To Yes – Has Begun TODAY
Posted by: Christine McSherry on March 01, 2014
#RaceToYes, #RDD14 #FDA
Yesterday was Rare Disease Day. Jenn, myself and other parents (The O’Brien family & James Raffone) siblings of those with Duchenne (Sarah Burgess) spent the day on Capital Hill – racing to every congressional office, all 535 offices – spreading the message of hope and the promise that eteplirsen could bring to our children. We were also joined by the Duchenne Alliance’s Scientific Advisor, Carlo Rago, PhD – who spoke to the historic moment that this drug will embark on once approved – first treatment in the most common of the dystrophinopathies, and the approval of an entirely new class of drugs that will change the face of medicine – forever. We all spoke in unison to every office visited, there is a treatment for Duchenne being evaluated by the FDA for Duchenne since late 2012, the FDA has not made any progress in approving this treatment and from that time, the science for this drug has only improved, the only thing that has declined or died – are those with Duchenne not on drug. By not approving this drug and utilizing an accelerated approval, we are blocking not only eteplirsen from getting to those who could benefit possibly by years, but many other potential therapies as well. We are in a “Race” for the FDA to say “Yes.” Our children can not wait another day. This is a perfect example of why FDASIA and PDUFA V were created – the FDA has had every opportunity to use the flexibility in this law, and they haven’t done it. Congressional members, please help.
Back home and in offices, our partners (Mindy Leffler, Tracy and Benjy Seckler, Marissa Penrod, and a team of dedicated, smart strategic colleagues) have been busy putting together the full campaign – “The Race To Yes.” While our feet and words were doing the work on the Hill, their quick minds and fingers have been diligently working around the clock creating a landscape where the community can gather, educate and gain traction to move eteplirsen (and soon other drugs) forward, faster. This group is creating a new voice in the community and among rare disease, helping achieve something even larger than we ever thought possible…pulling together patients/families with messaging that can not be ignored. A model that will continue to serve needs beyond eteplirsen, something none of us could have done without this team of dedicated, smart individuals.
Once, we felt alone, just as we did when our children were first diagnosed…as the weeks months and now years, have passed since Jenn first spoke out about eteplirsen, tremendous support has come – we know when progress is realized, and the path to treatments for Duchenne are changed, we will be in the company of so many others who helped and supported – and for that, we are forever grateful.
Again, we thank you for all that you have done. The Duchenne community and our supporters are helping to change history. Once again, we humbly ask for your help. Here is what you can do.
YOU’RE INVITED – JOIN THE RACE TO YES – Blog by Marissa Penrod
NOW LIVE – SIGN THE PETITION TO THE WHITE HOUSE
Twitter Handle: @TheRaceToYes
Instagram Name: TheRaceToYes
YouTube page: http://tinyurl.com/
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