PPMD's Blog – June 2016 Archive (5)

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…

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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 improve…

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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…

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Added by PPMD on June 20, 2016 at 9:30am — 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…

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Added by PPMD on June 8, 2016 at 12:00pm — 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…

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

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