News Update

[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.
July 20, 2016 | Learn more 

News Update

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.
July 19, 2016 | Learn more 

News Update - eNews

End Duchenne eNews: One Busy Summer

Catch up on the latest research and community updates in this month's End Duchenne eNews.
July 15, 2016 | Learn more 

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.
June 28, 2016 | Learn more 

News Update

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.
June 23, 2016 | Learn more 

News Update

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.
June 22, 2016 | Learn more 

News Update

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.
June 20, 2016 | Learn more 

News Update

[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.
June 11, 2016 | Learn more 

News Update

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.
June 10, 2016 | Learn more 

News Update

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.
June 9, 2016 | Learn more 

News Update

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.
June 8, 2016 | Learn more 

News Update

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.
June 1, 2016 | Learn more 

News Update

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!
May 26, 2016 | Learn more 

News Update

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.
May 25, 2016 | Learn more 

News Update

[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.
May 20, 2016 | Learn more 

News Update

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.
May 19, 2016 | Learn more 

Connect with PPMD
Facebook YouTube Twitter

Quick Links
How We Help

Our family-centered approach is at the heart of everything we do. Learn more.

Quick Links

Fund Research

More Links

PPMD Sites

new amazon banner

Continue to homepage