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. …Continue
Added by PPMD on July 28, 2016 at 2:18pm — No Comments
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…Continue
Added by Pat Furlong on July 25, 2016 at 12:00pm — No Comments
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.
Added by PPMD on July 21, 2016 at 11:25am — No Comments
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.
Added by PPMD on July 19, 2016 at 11:22am — No Comments
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!…Continue
Added by PPMD on July 14, 2016 at 1:36pm — No Comments
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…Continue
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…Continue
Added by PPMD on July 8, 2016 at 8:30am — No Comments
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…Continue
Added by PPMD on July 5, 2016 at 10:30am — No Comments