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





“Lucky” isn’t a word that usually comes to mind when thinking about children with Duchenne. The disease devastates kids and their families. It is 100% fatal. But, in a way, for the first time, we are fortunate. We have known what causes Duchenne for over 20 years.  Affected children do not produce a crucial protein called Dystrophin.  As a result, their muscles break down and they lose mobility and function and, ultimately, their lives.  Great minds in science have devoted their lives to cracking the code on Duchenne.  For decades, there have been necessary failures, massive community advocacy, grassroots and government funding, and numerous clinical trials.

It has been a long and grueling marathon.  But now, with a real potential treatment waiting for FDA approval, it has become a sprint.   A race to get this and future therapies to patients.  A Race to Yes.

Eteplirsen is the first Duchenne treatment with robust clinical data showing safety and efficacy, and consequently, we are faced with the miraculous yet sobering reality that this generation of Duchenne children could truly be the last to die or the first to survive Duchenne.  We are that close.  Our children deserve action. Days count. Hours count. Minutes count.  We have a real chance to cross the finish line. It’s time.

The month of February has been a remarkable one for Duchenne advocacy.  Several weeks ago, over 100 Congressional aides attended a briefing to learn about the urgency to approve Eteplirsen.  They heard from three of the world’s top Duchenne experts who agreed that this drug deserves accelerated approval.  “This drug works,” one expert stated. “It’s bleeding obvious,” added another.  Additionally, Congress again heard from our community this week about the critical importance of re-authorizing the MD Care Act.

We are fortunate to have champions in Congress. Congressman Roger Wicker is leading the charge on re-authorizing the MD Care Act.  Senator Ed Markey initiated a bipartisan Senate and House letter to the FDA urging the agency to make use of the law Congress passed (FDASIA) in 2012 that allows them to accelerate treatment approvals for diseases like Duchenne.

The momentum is real and the opportunity to save lives is within reach. So, today, in honor of Rare Disease Day, we are launching “The Race to Yes” on the web, on FB and on Instagram. This is our site, our place to unify efforts for one singular goal. This is our chance – to participate, to make an impact, to stay the course on The Race to Yes.
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TODAY: DO INSTAGRAM VIDEO or PHOTO

Record a video (see attached instructions), and/ or take a pic using the attached print-out sheet. hand-write your message on that piece of paper and take a pic/make a video and upload to Instagram.

 

ALWAYS use the following hashtags: #RaceToYes, #RDD14 and #FDA. NOTE: See attached Instagram messaging guide.

TODAY: DO FACEBOOK

Change your profile pic and cover photo with attached images

Make sure you “share the image that will be posted on the Race to Yes Facebook page: https://www.facebook.com/RacetoYes

Post photos on the “Race to Yes” Facebook page – so we can share your images as well.

 

Check out The Race to Yes Facebook page for graphics  to share throughout the day.

TODAY: TWEET

Always tweet to @TheRaceToYes and use the Instagram photos that you take – and the suggested messages that I sent over this afternoon – to drive the message out.

ALWAYS use the following hashtags: #RaceToYes, #RDD14 and #FDA.

Change Twitter profile to the image attached


NOW LIVE – SIGN THE PETITION TO THE WHITE HOUSE


NOW LIVE: Website: www.TheRaceToYes.org






Twitter Handle: @TheRaceToYes


Instagram Name: TheRaceToYes








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