February 18, 2025

Solid Biosciences Shares Initial Data from INSPIRE DUCHENNE Study of Gene Therapy Candidate SGT-003

Solid Biosciences Inc. has announced positive initial data from the company’s Phase 1/2 INSPIRE DUCHENNE trial evaluating SGT-003. SGT-003 is a gene therapy candidate for the treatment of Duchenne that delivers a microdystrophin via their…

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February 4, 2025

Cumberland Pharmaceuticals Announces Positive Results from Phase 2 FIGHT DMD Trial of Ifetroban for Treatment of Duchenne Cardiomyopathy

Cumberland Pharmaceuticals Inc. has announced positive top-line results from its Phase 2 FIGHT DMD trial evaluating ifetroban for the treatment of Duchenne cardiomyopathy. Ifetroban is an oral thromboxane receptor antagonist aimed at reducing inflammation and…

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January 27, 2025

Sarepta Therapeutics Announces Results from Part 2 of EMBARK Study of ELEVIDYS

Sarepta Therapeutics, Inc. has announced positive topline results from Part 2 of EMBARK, the company’s Phase 3 clinical study of ELEVIDYS. ELEVIDYS is a micro-dystrophin gene therapy used for the treatment of individuals aged four…

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January 23, 2025

Update from PPMD’s 2025 Duchenne Healthcare Professionals Summit

PPMD recently convened over 360 healthcare professionals, researchers, and industry partners, including representatives from the FDA, NIH, CDC, and international representatives in one room to discuss the latest updates in dystrophinopathy care and research at…

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January 17, 2025

Race to End Duchenne Kicks Off 20th Anniversary at Walt Disney World Marathon Weekend

The Race to End Duchenne program is celebrating our 20th anniversary in 2025, and we spent the weekend of January 8-12 kicking it off at the place where it all began – the Walt Disney…

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January 16, 2025

PPMD Advocacy in the 119th Congress and the 47th Administration

As the 119th Congress and the 47th Administration prepare to take office, PPMD is committed to furthering the needs of our community through comprehensive, creative, and tenacious advocacy on Capitol Hill, regardless of politics. Over…

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January 16, 2025

Advance the Research Evolution with The Duchenne Registry

Everyone can help advance the research evolution for Duchenne and Becker. Bringing data sources together from multiple research endeavors is a key component of PPMD’s plan to advance the research evolution, and The Duchenne Registry…

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January 15, 2025

PPMD Hosts 2025 Duchenne Healthcare Professionals Summit in San Diego, California

PPMD is excited to host the sixth in-person Duchenne Healthcare Professionals Summit in San Diego, California, January 15-17. Since the Summit’s inception in 2018, the event has brought together thousands of healthcare providers, researchers, leaders…

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January 10, 2025

Introducing PPMD’s Dystrophinopathy Clinical Research Network (DCRN): Advancing the Research Evolution

In our rapidly evolving therapeutic landscape, there is a critical need for cohesive infrastructure that helps track what happens when individuals are on more than one therapy and how we can continue to evolve standards…

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January 9, 2025

Avidity Biosciences Announces Plans to File Biologics License Application for Delpacibart Zotadirsen (AOC 1044)

Avidity Biosciences, Inc. has announced its plans for filing a Biologics License Application (BLA) with the U.S. Food and Drug Administration (FDA) for delpacibart zotadirsen (del-zota) for treatment of individuals living with Duchenne muscular dystrophy…

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