December 6, 2018

Wave Life Sciences Announces Positive Phase 1 Results for WVE-210201 in Duchenne Muscular Dystrophy

  Wave Life Sciences announced positive safety and tolerability results from the WVE-210201 Phase 1 clinical trial today. PPMD continues to be optimistic about the progress the team at Wave Life Sciences is making with…

Learn More

November 30, 2018

Support heart health–all gifts matched!

For the last two years, you have helped support PPMD’s Gene Therapy Initiative and because of your support, we are seeing real results, real potential, real hope. But with all the progress we have made…

Learn More

November 29, 2018

End Duchenne Tour Recap: Palo Alto, CA

PPMD headed to Palo Alto, CA for our End Duchenne Tour stop on November 3, 2018. Many families and members of the Duchenne community came together to learn about the latest information and resources for Duchenne families….

Learn More

November 29, 2018

An Update on Clinical Trial Innovation for Duchenne – a Master Protocol

PPMD is committed to making research and development of new medicines for families living with Duchenne as fast and efficient as possible.  Over the years, by listening to you, we have developed a clinical trials…

Learn More

November 28, 2018

PPMD Designates University of Virginia Children’s Hospital a Certified Duchenne Care Center

Today Parent Project Muscular Dystrophy (PPMD) named University of Virginia Children’s Hospital to be the 22nd clinic to be named a Certified Duchenne Care Center PPMD’s Certified Duchenne Care Center Program, which supports standardized, comprehensive care and services…

Learn More

November 21, 2018

Giving Thanks…for YOU!

As I write this, something is inevitably overcooking in my oven, the turkey may not be defrosted, and we are running low on Diet Coke. But amidst the craziness and chaos of the holiday, I…

Learn More

November 20, 2018

Santhera Enters into Agreement to Acquire Option for Exclusive Sub-License of Vamorolone

PPMD has learned that Santhera Pharmaceuticals and Idorsia Ltd have entered into an agreement under which Santhera will acquire the option to exclusively in-license vamorolone, currently being developed by Reveragen. Idorsia currently holds the license…

Learn More

November 19, 2018

PPMD 2018 Annual Conference – Polling Report

The 2018 PPMD Annual Conference provided a unique opportunity to conduct live polling of those joining us, including those joining in person and those joining virtually from home. The following report (download) provides the data…

Learn More

November 18, 2018

WATCH: Engaging Families in Care Guideline Development – Webinar Recording

On Wednesday, November 14, PPMD hosted a webinar on patient-centeredness of recently published endocrine care guidelines for Duchenne. Researchers from RAND Health Care and PPMD shared the results of their project that developed a new online…

Learn More

November 18, 2018

Slippery Rock Celebrates Coach To Cure MD with a Race to End Duchenne .1K

Slippery Rock University’s Coach To Cure MD game earlier this month was a memorable one! During halftime, three families were welcomed on the field with open arms to have their very first annual Race to End…

Learn More