There are thousands of children and young adults who are not getting the treatment they need. These individuals will decline over the next year waiting for treatments to get approved. They will lose the ability to walk, to feed themselves, and to hug their moms. The FDA is causing undue delay. They are not following process and are ignoring FDASIA, a law they helped Congress write. Congress should hold the FDA accountable and the FDA should show regulatory flexibility to provide the earliest possible access to treatment. Our advocacy work must continue in full force to get treatment for ALL with Duchenne.

We live in an incredibly hopeful time for those with Duchenne and rare diseases, but we need to continue to fight hard and make our voices heard, so safe and effective treatment gets to those who need it, as quickly as possible. Many with Duchenne, and other diseases, will decline while waiting for access to drugs that could help them. We need to stop this. I was featured in an article about it this week in U.S. News and World Report.

I need your support to stop the non-sense and get treatments more effectively. 

This month we kick off our end of the year giving campaign, I hope you will make a generous gift to save this generation from Duchenne and speed progress in treating rare diseases.