PPMD Community

July 2016 Blog Posts (8)

Every Single Family

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. …


Added by PPMD on July 28, 2016 at 2:18pm — No Comments

PTC Therapeutics Provides Regulatory Update on Translarna™

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…


Added by Pat Furlong on July 25, 2016 at 12:00pm — No Comments

Every Single Therapy

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.

Your sustaining gift to PPMD will help us continue to take a…


Added by PPMD on July 21, 2016 at 11:25am — No Comments

Every Single Connection

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.

Today, we are asking you to consider making a monthly,…


Added by PPMD on July 19, 2016 at 11:22am — No Comments

Thank you to our 2016 Connect Conference Resource Fair Providers!

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!…


Added by PPMD on July 14, 2016 at 1:36pm — No Comments

Santhera Updates on U.S. Regulatory Filing for Raxone® (idebenone) in Duchenne

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…


Added by PPMD on July 14, 2016 at 9:00am — 1 Comment

BioMarin Update for the Duchenne Community: July 7, 2016

BioMarin has provided a further update to the Duchenne Community following the company's announcement of the withdrawal of the EMA marketing application and discontinuation of clinical and regulatory development of drisapersen (BMN 051) and follow-on compounds BMN 044, BMN 045, and BMN…


Added by PPMD on July 8, 2016 at 8:30am — No Comments

PTC Initiates Phase 2 Clinical Trial of Translarna™ (ataluren) for Treatment of Nonsense Mutation Duchenne Muscular Dystrophy in Pediatric Patients Between the Ages of Two and Five Years

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…


Added by PPMD on July 5, 2016 at 10:30am — No Comments

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