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.
PPMD is undertaking multiple efforts to help improve clinical trial design in order to accelerate clinical trials and find promising therapies for Duchenne. But how does data aggregation really work? Our friends at D-RSC have spelled out some concepts and terms for you to make understanding all that PPMD is doing easier.
PPMD Names Children’s Mercy Hospital in Kansas City, MO, Certified Duchenne Care Center
Children’s Mercy Hospital in Kansas City, Missouri, became the 11th clinic named a Certified Duchenne Care Center by PPMD.
End Duchenne eNews: Every Single Carrier
Catch up on the latest research and community updates in this month's End Duchenne eNews.
PPMD Brings Together Experts to Assess Steroid Effects on Bone Health in Duchenne
More than 50 international experts in both pediatric and Duchenne bone health have gathered to attend PPMD’s Bone Health and Osteoporosis Workshop today & tomorrow in order to dive deeply into these important topics.
Every Single Carrier
PPMD is proud to formally announce our carrier initiative. Like everything we do, PPMD will look at carrier issues from a variety of angles, through a variety of lenses.
Changing the Paradigm: The Eteplirsen Ad Comm
There is no way around it, today was hard. Like you, we are dumbfounded by the closing votes and discussion. But a decision has not been made – these were recommendations. And we believe that the number of questions with mixed votes represent opportunities for us to change minds and continue to influence the agency.
April 25: FDA Advisory Committee Meeting for Eteplirsen - Live Webcast Information
The Center for Drug Evaluation and Research (CDER) plans to provide a free of charge, live webcast of the meeting.
PPMD Advocates Ensure Congress Recognizes Importance of Flexibility in FDA Reviews
Great news as Congress continues to communicate urgency to the FDA to apply flexibility in review of Duchenne therapies, thanks to our PPMD community!
PPMD Names Children’s Hospital Los Angeles 10th Certified Duchenne Care Center
PPMD is thrilled to announce our 10th Certified Duchenne Care Center -- Children's Hospital Los Angeles!
PPMD's Every Single [One] Tour Launched in Baltimore!
On Saturday, Duchenne community members from six states gathered together at Kennedy Krieger in Baltimore to hear the latest developments on clinical trials, clinical care practices, carrier programs, legislative and regulatory efforts, IEPs and school advocacy, physical therapy, genetic testing and more.
Senate Sends Strong Letter to FDA about Duchenne Therapies
With this letter now sent, both the House & Senate have communicated a strong message to the FDA about the need to use every possible tool at their disposal to speed access to promising Duchenne therapies.
PPMD’s Patient Preference Study about Pulmonary Outcomes – What we learned and why it matters
We are pleased to release the Community Report for this study, which takes a deeper look into the results. Later this year, an extended version of the report data will be published in multiple publications.
PPMD's 2016 Connect Conference Agenda Now Available
PPMD is excited to unveil the agenda for the 22nd Annual Connect Conference in Orlando, June 26-29. Make sure you register you and your family before the Early Bird Registration Rate ends April 15th!
ACTION ALERT: Please urge Your Senators to Sign Letter to FDA
Please urge your Senators to sign this important letter. Patients need and deserve therapeutic options and the FDA must use the tools they have been given to expedite these options.
How You Can Help with the Upcoming Ad Comm for Eteplirsen
We are just a few short weeks from the FDA Advisory Committee Meeting for eteplirsen on April 25th. The momentum is tremendous but we need YOU to take action leading up to the big day.
Webinar Recording: BMS Anti-Myostatin Adnectin Program
PPMD and Bristol-Myers Squibb (BMS) recently hosted a webinar to discuss the BMS Anti-Myostatin Adnectin Program.
PPMD Community Survey about the Akashi HT-100 study
If you or your child were in the Akashi HT-100 clinical trial that was recently suspended, please take our survey. Responses are needed by April 10th.
Update on Gene Replacement/Repair Strategies for Duchenne Muscular Dystrophy
There has been a flurry of press recently on gene therapy in Duchenne and CRISPR technology. With this barrage of information, PPMD wanted to take a deeper dive into gene therapy as a potential treatment for Duchenne.
PPMD is Initial Partner in OnPAR
PPMD is proud to participate in OnPar which provides access to peer-reviewed research proposals that directly relate to Duchenne research. OnPAR will provide us with an efficient way to solicit early stage and translational Duchenne research proposals, see the breadth of research in Duchenne, and gain access to novel research strategies.