End Duchenne eNews: Every Single Step
Catch up on the latest research and community updates in this month's End Duchenne eNews.
PPMD Sends Letter to FDA Regarding Duchenne Therapies
PPMD has sent a letter to the FDA requesting updates on various regulatory actions of tremendous importance to the Duchenne community.
[Webinar Recording] PTC Therapeutics Provides Regulatory Update on Translarna
PPMD recently hosted a webinar with PTC Therapeutics to discuss the latest news regarding Translarna and the path forward for the therapy with the FDA.
Help the Powers Family #TackleDuchenne!
The 9th Annual Coach To Cure MD on September 24 is shaping up to be our biggest year yet!
Duchenne mom Kim Powers shares why this is the year you should get in the game and help us #TackleDuchenne.
2016 Connect Conference Resources & Recordings
We are excited to share with you presentations from PPMD’s 22nd Annual Connect Conference! We will continue to update this page over the coming weeks with slides and video content.
[Webinar Recording] What is CRISPR/Cas9?
PPMD welcomed Dr. Eric Olson from UT Southwestern Medical Center to discuss the basics of gene editing and the potential permanent correction of Duchenne mutations with CRISPR/Cas9.
Every Single Connection
PPMD works tirelessly to arm you with information and resources through the PPMD Connect Conference, our Every Single One Tour meetings, or even virtual events like webinars, so that you can advocate for your child... so that your voice is heard.
PPMD Receives Grant from Marathon Pharmaceuticals to Help Support Acclaimed Certified Duchenne Care Center Program
The grant will enable PPMD to provide additional resources to improve patient care and patient recruitment for clinical trials through the centers.
Join the 2016 #PPMDConnect Conference Online!
Unable to attend this year's Annual Connect Conference in Orlando? Join us online! PPMD will be live streaming and our DuchenneConnect team will be live tweeting updates June 26-28.
PPMD Awards The Ohio State University Grant to Support Cardiomyopathy Therapy for Duchenne
Heart issues don't just affect some people with Duchenne; they affect all people with Duchenne. PPMD is so grateful to the work being done at institutes like The Ohio State University and we are proud to support Dr. Guttridge.
Understanding Gene Therapy & CRISPR/Cas9
Find out more about the latest in gene therapy, CRISPR/Cas9, and what these technologies could mean for Duchenne during three different opportunities this summer that promise to provide an in-depth analysis.