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.
End Duchenne eNews: One Busy Summer
Catch up on the latest research and community updates in this month's End Duchenne eNews.
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.
[Webinar Recording] MoveDMD: A Clinical Trial of Edasalonexent (CAT-1004) in Duchenne
PPMD and Dr. Joanne Donovan recently hosted a webinar to review information on edasalonexent (previously known as CAT-1004) and the MoveDMD trial.
Getting Results: Success from your Advocacy efforts in February!
Your actions this year have yielded incredible results for Duchenne related funding at the federal level.
PPMD Awards University of Washington Grant to Support Technology to Help Optimize Gene Therapy
PPMD is proud to support Dr. Odom from the University of Washington and we are hopeful that his work will help make gene therapy in Duchenne more effective and long lasting.
PPMD & BIO Release New Report on the Development of Patient Preference Studies
PPMD hopes that this report will provide guidance for companies seeking to incorporate the patient perspective at every stage of developing and delivering innovative new treatments.
BioMarin Discontinues Clinical Development of Drisapersen
BioMarin has announced that they were discontinuing the clinical development of drisapersen after interactions with both the FDA and EMA.
PPMD’s Every Single One Tour: Salt Lake City
With a successful Every Single One tour stop in Baltimore under our belts, PPMD visited Salt Lake City this past weekend. Thank you to all of the families from across Utah and Idaho who joined us!
Sarepta Announces FDA Will Not Complete the Review of the Eteplirsen NDA By The PDUFA Date
The FDA has notified Sarepta that they are continuing their review of eteplirsen and will not be able to complete their work by the PDUFA goal date of May 26.
[Webinar Recording] MissionDMD: FibroGen’s Anti-Fibrosis Program
PPMD and FibroGen recently hosted a webinar to discuss the launch of FibroGen's MissionDMD program that is being conducted to investigate FG-3019 in Duchenne.
Next Steps with Newborn Screening
PPMD is leading a national effort including more than 50 of the world’s top newborn screening and Duchenne experts to build a newborn screening infrastructure for Duchenne in the United States.