April 22, 2019

The Early Bird Rate for PPMD’s 2019 Annual Conference Ends Wednesday!

PPMD’S 2019 ANNUAL CONFERENCE JUNE 26 – JUNE 30, 2019 ORLANDO, FL Your last opportunity to join us at the 2019 PPMD Annual Conference at our Early Bird Registration Rate ends Wednesday, April 24! This…

Learn More

April 17, 2019

PPMD Awards $100,000 Grant to Nationwide Children’s Hospital to Further Explore GALGT2 Gene Therapy

PPMD is excited to announce that Drs. Paul Martin and Kevin Flanigan from the Research Institute at Nationwide Children’s Hospital have completed work under a $100,000 grant exploring GALGT2 gene therapy technology as a potential treatment…

Learn More

April 16, 2019

Wave Life Sciences Announces Suvodirsen Phase 1 Safety and Tolerability Data and Phase 2/3 Clinical Trial Design

Wave Life Sciences shared safety and tolerability data from their Phase 1 clinical trial evaluating investigational suvodirsen (WVE-210201) in patients with Duchenne. The results from this trial support progressing to a Phase 2/3 clinical trial,…

Learn More

April 15, 2019

Race Athlete Spotlight: Enzo Figueres

Enzo Figueres is an avid mountain biker but will be participating in his first road bike tour this May – with his son in a bike trailer! – as part of the Race to End…

Learn More

April 15, 2019

FibroGen Receives U.S. FDA Orphan Drug Designation for Pamrevlumab for the Treatment of Duchenne

FibroGen announced that FDA has granted Orphan Drug Designation for the company’s anti-CTGF antibody, pamrevlumab, for the treatment of people with Duchenne. According to their press release, all 21 non-ambulatory Duchenne patients in their ongoing…

Learn More

April 11, 2019

79 Members of Congress Send Letter of Strong Support for Duchenne Priorities for Next Year’s Budget

As a result of 143 meetings with Members of Congress in Washington DC, and over 2,000 action alert emails sent by this community, we are pleased to report a very successful outcome for the Duchenne…

Learn More

April 10, 2019

Unionville Run For Our Sons 5K Celebrates 10-Year Anniversary

Saturday, April 6 marked the 10th Annual Unionville Run For Our Sons 5K – a local DIY Race event hosted by the Johnson family benefiting Parent Project Muscular Dystrophy (PPMD) and our fight to end…

Learn More

April 8, 2019

Audentes Therapeutics Announces Expansion of AAV Technology Platform and Pipeline for Duchenne

Audentes Therapeutics announced that it is expanding its scientific platform and beginning development of new AAV-based genetic medicines for Duchenne. Audentes is a leading AAV-based genetic medicines company based in San Francisco. The company is…

Learn More

April 5, 2019

Catabasis Shares March 2019 Newsletters

Catabasis shared the latest edition of their community newsletter (also available in Spanish), including an update on their currently recruiting Phase 3 PolarisDMD trial for edasalonexent in Duchenne. Download the Catabasis newsletters: English Spanish

Learn More

April 4, 2019

End Duchenne Tour Recap: Omaha, NE

PPMD had the opportunity to travel to Omaha, NE on March 30 for our second End Duchenne Tour stop of 2019. We were joined by over 50 people including 18 families, 9 industry partners, and 10…

Learn More