PPMD Announces Gene Therapy Policy Forum
PPMD, in collaboration with the FDA’s Center for Biologics Research and Evaluation (CBER), the agency division responsible for reviewing gene therapies, is excited to announce a Gene Therapy Policy Forum taking place during PPMD's 2016 Advocacy Conference.
Growing Our Team
The New Year has brought new changes to the PPMD family. We ended 2015 bidding a fond farewell to Dr. John Porter who spent the last year as part of the PPMD team, and last week we welcomed Abby Bronson who will be taking on the role of Senior Vice President of Research Strategy.
PPMD and a coalition of Duchenne foundations continue to work together on preparing for the Sarepta Ad Comm. As of today, we are still waiting on a new confirmed date. With the postponement of the Ad Comm, the Congressional letter to FDA remains open. Has your House Members signed on yet?
FDA Ad Comm for Eteplirsen Postponed Due to Inclement Weather
We are deeply saddened, disappointed, and distressed to report that the highly-anticipated Ad Comm for eteplirsen has been postponed due to the blizzard anticipated to hit DC on Friday. We are grateful to our extraordinarily powerful Duchenne community who has been willing to travel into the eye of this storm to fight Duchenne. Our community has proven once again to be a force stronger than any winter storm, and much stronger than Duchenne itself. As soon as we receive any updates regarding a new date for the Ad Comm, we will let you know.
Webinar Recording: HOPE-Duchenne Trial - Capricor Therapeutics
Last week, PPMD, Coalition Duchenne, and Capricor hosted a webinar to discuss the HOPE-Duchenne trial which recently opened for enrollment.
Marathon Expanded Access Program for Deflazacort
There have been questions about whether taking Marathon’s deflazacort would impact involvement in currently ongoing and/or future clinical trials. We have had the opportunity to talk both with Marathon Pharmaceuticals, as well as several other industry partners, about the use of Marathon’s deflazacort.
Urge House Member - Apply FDASIA Tools to Duchenne
There has never been a moment like this in our Duchenne community. Today the FDA released briefing documents on the application for eteplirsen. It is clear that we continue to face a serious regulatory challenge as a community.
End Duchenne eNews: New Year, New Hope
Catch up on the latest research and community updates in this month's End Duchenne eNews.
A Blow to Our Community – But Our Work Continues
Today our community was dealt a painful blow as the FDA issued a Complete Response letter to BioMarin regarding the company’s New Drug Application (NDA) for Kyndrisa (drisapersen). As stated in BioMarin’s press release, “The FDA issues Complete Response letters to indicate that the review cycle for an application is complete and that the application is not ready for approval in its present form. FDA has concluded that the standard of substantial evidence of effectiveness has not been met.”
PPMD Responds to Revisions to “The Common Rule”
The Common Rule or the 'Federal Policy for the Protection of Human Subjects' is the policy that determines how you are consented when participating in trials and research, what types of research are considered to be ethical, and how biomedical samples acquired from patients can be used and stored.
Dysphagia in Duchenne: practical recommendations to guide management
Dysphasia, or difficulty swallowing, effects many patients with Duchenne. Complaints of "something stuck in my throat" are not uncommon. A new article on dysphagia in Duchenne presents a clear step by step plan of care.
Webinar 1/13/16: HOPE-Duchenne Trial - Capricor Therapeutics
Please join PPMD and Capricor as we host a webinar Wednesday, January 13 at 1pm eastern to discuss the HOPE-Duchenne trial which recently opened for enrollment in the US.
We did it! Over $270,000 raised to support early-stage research.
Last month PPMD told you we hoped to raise $200,000 to support new, early-stage research. And how did you respond? By not only hitting our goal, but surpassing it!
Finding Hope in CRISPR/Cas9
Duchenne made national headlines over the holidays thanks to the latest news on gene editing technology. PPMD wanted to help take a deeper dive into gene editing and CRISPR/Cas9 so that we can better understand the potential of this cutting-edge technology.
Last chance to have your donation doubled!
Just a few hours left to match every single gift! We’re so close to hitting our goal—but we need to raise $24,000 more by midnight tonight. Help us take full advantage of the matching gift by making a tax-deductible donation before midnight!