March 4, 2025

Capricor Therapeutics Announces FDA Acceptance and Priority Review of Biologics License Application for Deramiocel

PPMD is excited to learn that the FDA has accepted Capricor Therapeutics’ Biologics License Application (BLA) for review, seeking traditional approval for deramiocel (CAP-1002). Additionally, the FDA granted the BLA Priority Review, reducing the review…

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February 28, 2025

WATCH: Solid Biosciences – Update on Gene Therapy Candidate SGT-003 (Webinar Recording)

Members of the Solid Biosciences team recently joined PPMD for a community webinar to review initial positive data from the INSPIRE DUCHENNE study of SGT-003, a next-generation microdystrophin gene therapy candidate. The Solid team provided…

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February 24, 2025

Entrada Therapeutics Announces FDA Removal of Clinical Hold on ENTR-601-44

Entrada Therapeutics, Inc. has announced that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold on ENTR-601-44 and provided authorization to initiate ELEVATE-44-102, a Phase 1b multiple ascending dose clinical study of…

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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 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 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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January 2, 2025

Capricor Therapeutics Completes Submission of Biologics License Application to the U.S. FDA for Deramiocel

PPMD is excited to learn that Capricor Therapeutics has completed the submission of its rolling Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) seeking full approval for deramiocel (CAP-1002) for the…

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