In the 20+ years PPMD has been around, the average lifespan of people with Duchenne has increased. This incredible leap is not because of any drug interventions or treatments because as we all know, to date there are no approved therapies. Rather this is because of the push from PPMD to advance care over the last two decades.
PPMD strives to ensure that every single person with Duchenne receives optimal care. …Continue
Added by PPMD on July 28, 2016 at 2:18pm — No Comments
PTC recently participated in discussions with FDA to discuss the Refuse to File (RTF) letter issued on February 22, 2016 with respect to the company's New Drug Application (NDA) for Translarna for the treatment of Duchenne. Today PTC…Continue
Added by Pat Furlong on July 25, 2016 at 12:00pm — No Comments
At the heart of PPMD’s mission lies our unwavering commitment to seek out, fund, and nurture the most promising Duchenne research – more specifically, therapies with the potential to help every single diagnosis.
Added by PPMD on July 21, 2016 at 11:25am — No Comments
For more than two decades, PPMD has helped to change the landscape of Duchenne in every area – research, care, advocacy, and awareness. You have made this possible. Every single person that supports PPMD gives us the resources we need to have the greatest impact, to attack Duchenne from every angle.
Added by PPMD on July 19, 2016 at 11:22am — No Comments
The 2016 Connect Conference again provided a forum for families to interact with a variety of resource providers. We thank our resource fair sponsors for working with us to make every single resource known to our community!…Continue
Added by PPMD on July 14, 2016 at 1:36pm — No Comments
PPMD is deeply disappointed with the FDA’s conclusion regarding Santhera's proposed subpart H (Accelerated Approval) for Raxone (idebenone). Because individuals in the study were not taking steroids, FDA has recommended Santhera complete the SIDEROS study on individuals using…Continue
PTC Therapeutics has announced that they have initiated a Phase 2 clinical trial of Translarna™ (ataluren) for children 2 to 5 years old. Details of site locations, etc. are on clinicaltrials.gov. Below is the announcement…Continue
Added by PPMD on July 5, 2016 at 10:30am — No Comments
Parent Project Muscular Dystrophy (PPMD) announced today plans to award Dr. Denis Guttridge of The Ohio State University with a $48,000 grant for his work in cardiac issues in Duchenne. Duchenne affects muscles, and since the heart is a muscle too,…Continue
Added by PPMD on June 22, 2016 at 10:30am — No Comments
BioMarin is providing a further update to the Duchenne Community in light of recent announcements of withdrawal of the EMA marketing application and discontinuation of clinical and regulatory development of drisapersen (BMN 051) and follow-on products BMN 044, BMN 045 and BMN 053.
Is there any Update…
Added by PPMD on June 20, 2016 at 9:30am — No Comments
Genetic counselors, whether they are part of PPMD's DuchenneConnect team or part of your local care team, are an excellent resource to consider when you have questions about genetic test results for Duchenne and Becker muscular…Continue
You’ve seen the headlines and heard the name, now find out more about the latest in gene therapy, CRISPR/Cas9, and what these technologies could mean for Duchenne during three different opportunities that promise to provide an in-depth analysis:
Added by Abby Bronson on June 16, 2016 at 11:04am — No Comments
Our Duchenne community’s life-saving research efforts are funded through a number of public and private sources. Beginning in FY 2011, the federal funding to Duchenne began to include the Congressionally Directed Medical…Continue
Added by Annie Kennedy on June 16, 2016 at 11:00am — No Comments
Thanks to your advocacy, this week we learned that all of the Duchenne priorities we advocated for this year with Members of Congress are one step closer to being included in next year’s budget.
Whether you traveled to DC for face-to-face meetings with members, or advocated from…Continue
Added by Ryan Fischer on June 10, 2016 at 12:00pm — No Comments
The promise of gene therapy continues to build momentum, in Duchenne and also in other conditions. Much has been learned about the immunological and delivery challenges of the approach since gene therapy’s early attempts in the 1990s.
In Duchenne, some of the early work in gene therapy ended with immune system reactions to the new dystrophin that was expressed by the transfected gene. But this was not…Continue
Added by Abby Bronson on June 9, 2016 at 12:00pm — No Comments
Today, the Biotechnology Innovation Organization (BIO) and Parent Project Muscular Dystrophy (PPMD) released a new report, “Key Considerations for Developing and Integrating Patient…Continue
Added by PPMD on June 8, 2016 at 12:00pm — No Comments
As you know, PPMD has been helping to lead national advocacy efforts around the 21st Century Cures/ Senate Innovation legislation for more than two years and we now have a very short window of opportunity in which the…Continue
Added by Annie Kennedy on June 7, 2016 at 1:00pm — No Comments
Santhera Pharmaceuticals announced today that additional data from the pivotal phase III trial (DELOS) were published online as an article in press in…Continue
Today is June 1 and I know many of us feel like 2016 has beat up this community already. With every small victory, we face another major setback.
As you have heard by now,…Continue
With a successful ESO tour stop in Baltimore under our belts, PPMD visited Salt Lake City this past weekend! There’s a special sense of…Continue
Added by Kathi Kinnett on May 26, 2016 at 3:00pm — No Comments
The FDA has notified Sarepta that they are continuing their review of eteplirsen and will not be able to complete…Continue
Added by PPMD on May 25, 2016 at 12:00pm — No Comments