PPMD is excited to today announce a collaboration with BioMarin Pharmaceutical Inc., PTC Therapeutics, and Sarepta Therapeutics, Inc. in the next phase of the highly successful Decode Duchenne program. Decode Duchenne is a nationwide program to assist individuals with…

On September 2nd, PPMD convened a public webinar for the Duchenne community focused on FDA Advisory Committee Meetings (or “Ad Comms”). After working closely with representatives from FDA, we are excited to share some of the frequently asked questions that arise regarding the Ad Comm process, as well as some specifically relevant to our community. As we get closer to possible dates for these meetings, we will add information as it becomes available.…

We are thrilled to announce that the Advancing Targeted Drugs for Rare Diseases Act bill dropped in the Senate today (see press release below)! PPMD has been working with the Senate HELP Committee and Sarepta for the last several months on this 21st Century Cures provision which morphed into stand-alone legislation (view PPMD's most updated endorsement of this…

On the eve of the 2nd Annual World Duchenne Awareness Day, PPMD is excited to be one of five recipients of PTC Therapeutics’ STRIVE Awards! This $30,000 grant will be used to help PPMD launch a campaign for…