June 2016 Blog Posts (12)

Sarepta Update for the Duchenne Community Regarding ESSENCE Study

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…


Added by PPMD on June 24, 2016 at 9:00am — No Comments

PPMD Awards The Ohio State University Grant to Support Cardiomyopathy Therapy for Duchenne

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, cardiac problems remain a major concern for patients. PPMD has spent the last several years supporting efforts to…


Added by PPMD on June 22, 2016 at 10:30am — No Comments

BioMarin Update for the Duchenne Community

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 Testing for Duchenne & Becker: When Is Re-Testing Needed?

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 dystrophy.

Today, I’d…


Added by Lauren Bogue on June 16, 2016 at 12:00pm — 1 Comment

Understanding Gene Therapy & CRISPR/Cas9

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 Cas9: Similarities and differences, Tuesday June 28…

Added by Abby Bronson on June 16, 2016 at 11:04am — No Comments

PPMD Responds to Threat to Lose DOD Funding

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 Program (CDMRP) through the Department of Defense.…


Added by Annie Kennedy on June 16, 2016 at 11:00am — No Comments

Getting Results: Success from your Advocacy efforts in February!

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 through emails, phone calls, and social media – your actions…


Added by Ryan Fischer on June 10, 2016 at 12:00pm — No Comments

PPMD Awards University of Washington Grant to Support Technology to Help Optimize Gene Therapy

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…


Added by Abby Bronson on June 9, 2016 at 12:00pm — No Comments

PPMD & BIO Release New Report on the Development of Patient Preference Studies

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


Added by PPMD on June 8, 2016 at 12:00pm — No Comments

National 21st Century Cures Act/ Senate Cures Day of Advocacy - TODAY!

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 can act to finalize this legislation and then both chambers…


Added by Annie Kennedy on June 7, 2016 at 1:00pm — No Comments

New Data from Santhera’s Phase III Trial (DELOS) in Duchenne Published in Neuromuscular Disorders

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


Added by PPMD on June 1, 2016 at 12:00pm — 1 Comment

BioMarin Discontinues Clinical Development of Drisapersen

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,…


Added by Pat Furlong on June 1, 2016 at 9:57am — 2 Comments

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