In an effort to provide regular communication on BioMarin's Duchenne muscular dystrophy programs to the Duchenne community, the company is circulating a quarterly update. This update is the first of its kind for BioMarin, and they welcome your feedback to this new communication channel, which you may do by contacting BioMarin Patient Advocacy (contact details are…

Summit Therapeutics plc announced that its Phase 1b modified diet clinical trial of SMT C1100 for the treatment of Duchenne met its primary objective with half of the patients who received the higher dose of SMT C1100 achieving desired plasma levels while following specific dietary guidance.  Based on these results, Summit will advance SMT C1100 into a Phase 2 open-label clinical trial. Congratulations to our friends at Summit and we look forward to hearing more about next…

Last week, Marathon Pharmaceuticals announced that the company will begin the New Drug Application (NDA) process for deflazacort as a potential treatment for patients with Duchenne following a positive pre-NDA meeting with FDA.  The…

We are coming to the end of the 60 day open comment period for the FDA’s draft guidance on Duchenne. PPMD submitted our comments to the agency yesterday –…

Call and email your Senators today! The Food and Drug Administration’s efforts to support meaningful patient engagement, also known as Patient-Focused Drug Development (PFDD), includes the strengthening of existing tools and creation of new ones needed to fill gaps, policies to allow for greater engagement of external experts in the review process, and a…