Sarepta has shared an update for the Duchenne community regarding recent changes to the Phase III confirmatory clinical study, ESSENCE (Study 4045-301), as posted on clinicaltrials.gov [identifier: NCT02500381]. Sarepta acknowledges concerns regarding a 96 week…

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,…

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…

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…

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:

• Gene Therapy and CRISPR…

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 Research…

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 home…

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…

Today, the Biotechnology Innovation Organization (BIO) and Parent Project Muscular Dystrophy (PPMD) released a new report, “Key Considerations for Developing and Integrating Patient…

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 Senate…

Santhera Pharmaceuticals announced today that additional data from the pivotal phase III trial (DELOS) were published online as an article in press in…

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,…