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
Added by Lauren Bogue on June 16, 2016 at 12:00pm — No Comments
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
PPMD Awards Eleventh Clinic Certification as Part of Effort to Ensure People with Duchenne Receive Optimal Care
Added by PPMD on May 16, 2016 at 2:08pm — No Comments
A couple of months ago we posted a write up about some of the efforts PPMD is undertaking to help improve…Continue
For more than two years, PPMD has been leading a national effort to build a newborn screening infrastructure for Duchenne in the U.S.
Led by PPMD's Annie Kennedy, Michele Lloyd–Puryear, M.D., Ph.D., Newborn Screening Consultant for PPMD, and Jerry Mendell, MD of Nationwide Children's, the…Continue
Added by PPMD on May 16, 2016 at 8:30am — No Comments
Added by Kathi Kinnett on May 12, 2016 at 9:00am — No Comments
I am a genetic carrier of Duchenne. My mother was not. This means I was one of the 1:4600 females born with this genetic mutation. As a child, I had muscle cramps, ‘growing pains’ my parents would say. And from those ‘growing pains,’ 20 years later, my sons were diagnosed and I first learned…Continue
It's been a rough few weeks for this community, but our efforts to shape and reshape access barrier issues for our community continue -- and last Wednesday we took a giant leap forward. It was a pivotal day that I was proud to be a part of!
The federal intra-agency…Continue
There is no way around it, today was hard. Hundreds of us – the largest gathering of Duchenne families in history – came together to tell the FDA that eteplirsen is safe, effective, and should be approved using the tools…Continue