February 11, 2021

A DIY Virtual Walk Success Story

Rally4RJ Virtual Walk in the Park: A DIY Virtual Walk Success Story! Suzanne Sullivan and her family were excited to hold their 2nd annual Rally4RJ 1 Mile Walk in the Park in the summer of…

Learn More

February 10, 2021

“See you on the Road”: PPMD’s End Duchenne Tour 2021 Stops

Stemming from our PPMD’s Connect Roundtables, PPMD’s End Duchenne Tour stops were formally established in 2015 as the Every Single One Tour. Tour stops are typically one-day long events, providing updates on the latest clinical…

Learn More

February 10, 2021

Gene Therapy and Immune Response: Understanding Challenges and Strategies

PPMD recently announced the formation of the Pediatric Gene Therapy and Medical Ethics (PGTME) Working Group to address the challenges involved in gene therapy. As part of their recent educational series, the PGTME held a…

Learn More

February 9, 2021

PPMD Receives $100,000 Award from Duchenne Research Fund to Address Neurobehavioral Issues in Duchenne

Parent Project Muscular Dystrophy (PPMD) is excited to announce sponsorship from the Duchenne Research Fund for an award totaling over $100,000. As part of PPMD’s new Brain Initiative, the award will support work in understanding…

Learn More

February 5, 2021

PTC Shares Results of Study 045 of Translarna™ (Ataluren) in Patients with Nonsense Mutation Duchenne

Twenty years ago, we were first introduced to PTC Therapeutics. We watched as PTC-124 was in early studies of safety and dose finding. Over the years, as studies continued, PTC-124 was referred to as ataluren…

Learn More

February 4, 2021

WATCH: Planning For Your Future – Navigating Benefits & Finances

Last week we were joined by Amber Sapp, parent, and Leona Filis, attorney, to discuss planning for your family’s future in regards to benefits and finances. Although these subjects can feel overwhelming, Amber and Leona…

Learn More

February 3, 2021

TODAY: Virtual Town Hall at 1 PM EST

Advocacy in Action: Virtual Town Hall Facebook Live | February 3, 2021 To commemorate the passage of the MD-CARE Act, PPMD will host a Virtual Town Hall on Wednesday, February 3, 1 PM – 4…

Learn More

February 1, 2021

Santhera Shares Community Update on Phase 2b Trial for Vamorolone

Santhera Pharmaceuticals has shared an update with the community regarding the phase 2b trial for vamorolone, which the company expects to have data from in the second quarter of 2021. The update includes contact information…

Learn More

January 29, 2021

20 Years of the MD-CARE Act: Commemorating Progress & Charting the Future Course

On February 27, 2001, 20 years ago, the Duchenne community gathered in Washington, DC for the first ever Senate hearing focused on Duchenne muscular dystrophy. On that much-anticipated day, a young teenager, Ben Cumbo, Goodwill…

Learn More

January 26, 2021

COVID-19 Vaccination & Duchenne | Priority Access – Sample Letter

As states continue to move through their Phase 1 COVID-19 vaccination distribution protocols, PPMD wants to make sure you are equipped with resources to advocate for priority access. For this reason, we have developed a…

Learn More