November 14, 2025

FDA Approves New Safety Warning and Revised Indication for ELEVIDYS

Today the FDA announced a significant update to the labeling and indication of ELEVIDYS for the treatment of individuals living with Duchenne following reports of death based on acute liver failure (ALF) in non-ambulatory patients…

Learn More

November 14, 2025

Protected: PPMD Continues to Drive Progress with Your Support

There is no excerpt because this is a protected post.

Learn More

November 10, 2025

Tips for Smooth Holiday Travel

The holidays are a special time to connect with family and friends, and for many, that involves travel. To help make your journey a little easier, PPMD has gathered some helpful travel tips and resources!…

Learn More

November 7, 2025

Welcoming the New Members of PAAC for Fall 2025

PPMD’s Adult Advisory Committee (PAAC) recently expanded with 19 inspiring new members, bringing the committee to its largest size ever—73 members strong. These incredible individuals bring diverse academic backgrounds ranging from marketing and communication to…

Learn More

November 6, 2025

Advancing Brain and Behavior Care in Dystrophinopathy

Recently, PPMD led a meeting with experts from across the US and Europe to discuss the evolving understanding of brain and behavior implications in individuals with dystrophinopathy. The two day workshop, led by Drs. Molly…

Learn More

November 5, 2025

Understanding Fat Embolism Syndrome (FES) and Protecting Bone Health

PPMD recently hosted a webinar about Fat Embolism Syndrome (FES), covering what FES is, what symptoms to watch for, and how to help keep people with dystrophinopathy safe and healthy. Four clinical experts shared practical…

Learn More

November 5, 2025

Solid Biosciences Shares Interim Data from INSPIRE DUCHENNE Trial of SGT-003

Solid Biosciences Inc. has announced positive new interim data from the Phase 1/2 INSPIRE DUCHENNE clinical trial and provided an update on its planned meeting with the U.S. Food and Drug Administration (FDA) to discuss…

Learn More

November 4, 2025

Sarepta Announces Completion of ESSENCE Trial for Exon 45 & 53 Skipping Therapies

Sarepta Therapeutics has announced top-line results from the ESSENCE trial, a global, randomized double-blind, placebo-controlled Phase 3 confirmatory study evaluating the efficacy and safety of AMONDYS 45 (casimersen) and VYONDYS 53 (golodirsen) in individuals living…

Learn More

November 4, 2025

PPMD Provides $400,000 in Funding to MyoGene Bio Through PPMD Venture Pathways Program to Support Development of Gene Editing Platform

PPMD and MyoGene Bio (MyoGene) are excited to announce that PPMD has provided $400,000 in funding to MyoGene through PPMD Venture Pathways, the organization’s venture-philanthropy initiative that provides industry funding to accelerate therapeutic development for…

Learn More

October 27, 2025

Avidity Biosciences to be Acquired by Novartis

Yesterday, Avidity Biosciences announced a definitive agreement to be acquired by Novartis. The transaction includes Avidity’s late-stage programs, including delpacibart zotadirsen (del-zota) for Duchenne. According to Avidity’s announcement, the company and Novartis intend to keep…

Learn More