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 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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December 19, 2024

WATCH: Sevasemten – CANYON Trial Topline Results (Webinar Recording)

Edgewise Therapeutics recently joined PPMD for a breaking news community webinar, during which Edgewise provided an update on the recently released topline results from its CANYON double-blind, randomized, placebo-controlled design to investigate the effect of…

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December 18, 2024

Percheron Announces Termination of Phase 2B Study of Avicursen for Duchenne

PPMD is disappointed to learn of Percheron Therapeutics’ recent decision to terminate the company’s ongoing Phase 2b randomized, placebo-controlled trial of avicursen (ATL1102) in non-ambulatory individuals living with Duchenne following the release of topline six-month…

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December 16, 2024

PepGen Announces Clinical Hold in the U.S. on IND Application to Initiate CONNECT2-EDO51 Phase 2 Study of PGN-EDO51

PPMD is disappointed to learn that PepGen Inc. has received a full clinical hold notice from the U.S. Food and Drug Administration (FDA) regarding its Investigational New Drug (IND) application to initiate the CONNECT2-EDO51 clinical…

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December 16, 2024

Edgewise Therapeutics Announces Positive Topline Results from CANYON Phase 2 Trial of Sevasemten in Individuals with Becker

Edgewise Therapeutics, Inc., has announced positive topline results from the double-blind, randomized, placebo-controlled Phase 2 CANYON trial of sevasemten in individuals with Becker muscular dystrophy. Sevasemten is an orally administered small molecule inhibitor designed to…

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November 26, 2024

Equitable Access in Clinical Trials—A Call for Action and Collaboration

Living with Duchenne and Becker presents unique challenges, especially for families from underserved or marginalized communities. Health disparities, delayed diagnoses, and limited access to care and clinical research create barriers that often result in diminished…

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November 21, 2024

WATCH: RGX-202 – AFFINITY DUCHENNE Pivotal Program and Functional Data (Webinar Recording)

REGENXBIO recently joined PPMD for a community webinar to provide an update on its AFFINITY DUCHENNE trial of RGX-202, REGENXBIO’s microdystrophin gene therapy. Dr. Naz Dastgir discussed the company’s pivotal trial plans and shared interim…

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