When we launched Team Joseph five years ago, I anticipated major hurdles. I thought the science might not be advanced enough to lead to new medicines in time to save Joseph’s life. I thought we might have trouble raising enough money to fund crucial medical research and drug development.
But never in a million years did I think we would face this: There is a drug that is safe and effective in a small clinical trial that has lasted more than two years. Some of the boys taking the medicine are even IMPROVING, a word that is never uttered in relation to Duchenne. Yet the drug is only available to the 12 kids in the trial, and it could be YEARS before other kids with Duchenne are allowed access. The one thought that comes to mind is, “You have got to be kidding me.”
You can help us change this NOW. If you’re in a rush, skip to the last paragraphs. If you have 3 minutes, continue here:
Many of you have written about this travesty to the FDA. Now it’s time to step it up a notch. Please ACT TODAY and contact your Congressmen and Senators. On Friday, February 7 at 10 a.m., there will be an important briefing on Capitol Hill for Congressional staff on a pending decision by the FDA on whether to proceed with Accelerated Approval for this treatment for Duchenne muscular dystrophy.
The briefing will feature a panel of renowned DMD researchers and scientists, as well as our government affairs team, who will brief staffers on how they can help. We need YOUR HELP to ensure your Congressmen and Senators send their health policy staffers to this important briefing. (You do not need to worry about coming yourself as we will have parent representatives there and will update the community directly after the briefing.)
A similar briefing held last year resulted in over 30 Members of Congress writing to the FDA on behalf of our children. While there are many issues of concern to our community, we think we can all agree we need to continue to engage our elected leaders on this timely and urgent matter.
Eteplirsen is a treatment for DMD that has been in a Phase II clinical trial for more than 120 weeks. Children with Duchenne participating in the trial have NOT experienced adverse clinical effects from the treatment – which is evidence of its safety. At the same time, they have experienced stabilization in their condition that is remarkable and unseen even among the most experienced clinicians in the field. In other words, it is safe and it works!
The FDA’s upcoming decision is extremely significant. It not only affects this particular treatment, but has tremendous implications for new treatments that are coming down the pike. Joseph will not benefit from Eteplirsen, as it is a treatment specific to a child’s mutation that causes Duchenne, but he may be helped by a similar medicine the company will develop once this first drug blazes the path toward approval.
LAST PARAGRAPHS: To help, please send an e-mail to DMDaction@gmail.com and provide your name and 9-digit zip code. We will respond promptly with a draft e-mail you can send to your Congressmen and Senators to request their attendance.
Thank you — thank you — thank you — while I know we frequently ask our supporters for help, together, we’re trying to save lives, and we are too far along to stop now!!! So please, send that email to DMDaction@gmail.com TODAY.
**To look-up your 9-digit zip code, please click here.