Yesterday, I had the distinct privilege of representing PPMD and our Duchenne community as I sat before a full room of Senate staffers and congressional aides in the Dirksen Senate office building. Our goal was to urge them to press forward on the Innovation for Healthier Americans bill (also referred to as the ‘Innovation…

In the last few weeks we’ve heard the news from several companies working to develop therapies for Duchenne that they have received Rare Pediatric Disease Designations from the FDA. Santhera, Sarepta, Catabasis, and Biomarin.

We as a community have celebrated this news. But what does it actually mean?

And did you know that it…