By February 1, 2014November 27th, 2018Tracy's Blog
Let them grow old together!













We have a problem, one that I never expected us to face. When we launched Charley’s fund nine years ago, I anticipated major hurdles. I thought the science might not be advanced enough to lead to new medicines in time to save Charley’s life. I thought we would have trouble raising enough money to fund crucial medical research and drug development. I worried that Charley might go through an ugly stage and I wouldn’t be able to splash adorable pictures all over our website. But never in a million years did I think we would face this: We have 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. My kids hate when I use their lingo, but I can’t think of a grown-up phrase that says it as well as this: WTF?

Let’s change this NOW. If you’re in a rush, skip to the last paragraph. 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 10:00am, there will be an important briefing on Capitol Hill for Congressional staff on a pending decision by FDA on whether to proceed with Accelerated Approval for treatments 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 boys. While there are many issues of concern to our community, we think that we can all agree that 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 over 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. Charley cannot benefit from Eteplirsen, but he can be helped by a similar medicine the company will develop once this first drug blazes the path toward approval.

LAST PARAGRAPH: To help, please send an e-mail to 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.

**To look-up your 9-digit zip code, please click here.

Thank you — thank you — thank you — yes I am tired of asking you for more help and I am personally exhausted but we’re too far along to stop now!!! So please send that email to TODAY.