Blog

Calling All Men With Becker – This Imaging Study is for You! (Webinar Recording & Resources)

PPMD was happy to recently host Dr. Krista Vandenborne, Director of ImagingNMD, and Dr. Claudia Senesac, ImagingNMD project manager, from University of Florida’s ongoing ImagingNMD program for a webinar on the development of a new…

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WATCH: Carrier Cardiac Care (Webinar Recording)

PPMD hosted a webinar on March 22, 2022 to discuss cardiac care for female carriers of Duchenne and Becker. Panelists discussed the importance of carrier health, learnings from the PPMD-sponsored carrier study at Nationwide Children’s…

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Santhera and ReveraGen Start Rolling NDA Submission to the FDA for Vamorolone for the Treatment of Duchenne

Santhera and ReveraGen have announced the initiation of a rolling new drug application (NDA) submission to the U.S. Food and Drug Administration (FDA) for vamorolone for the treatment of Duchenne. This is the first step…

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Santhera and ReveraGen Present Efficacy and Safety Data with Vamorolone in Duchenne

Santhera and ReveraGen have shared an update on the evidence of clinical efficacy and safety of vamorolone in participants with Duchenne, based on the positive outcome of the pivotal Phase 2b VISION-DMD study and additional Phase…

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Solid Biosciences Reports 2-Year Efficacy and Safety Data from Ongoing Phase I/II IGNITE DMD Clinical Trial of SGT-001

Solid Biosciences has shared an update from IGNITE DMD, an ongoing Phase I/II clinical trial evaluating its gene therapy candidate SGT-001 in participants with Duchenne. In the announcement, Solid reports positive two-year efficacy and safety…

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The Lancet Publishes Positive Results from Capricor’s Phase 2 Study of CAP-1002 in Late-Stage Duchenne

Capricor Therapeutics has announced that The Lancet has published positive results from the company’s HOPE-2 Phase 2 clinical trial evaluating CAP-1002 in advanced stages of Duchenne. Findings from the study demonstrated that young men in the advanced stages…

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PPMD’s Certified Duchenne Care Center Program – 2021 Impact & Progress

Every single person living with Duchenne deserves the best care possible. It is important that families know who is in charge of delivering the care they need and understand what centers are providing optimal, standardized…

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February 4, 2022

WATCH: FibroGen DMD Pamrevlumab Phase 3 Clinical Studies Highlight (Webinar Recording)

FibroGen joined PPMD for a webinar on February 2, 2022. During the webinar, FibroGen discussed the clinical rationale for the use of Pamrevlumab by highlighting its Phase 2 study in non-ambulatory participants with Duchenne and…

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January 25, 2022

Capricor & Nippon Shinyaku Enter Partnership for Commercialization and Distribution of CAP-1002 in the U.S.

Capricor Therapeutics and Nippon Shinyaku (US subsidiary: NS Pharma) have announced that they have entered into an agreement for the exclusive commercialization and distribution in the United States of CAP-1002, Capricor’s novel cell therapy candidate…

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January 20, 2022

Dyne Therapeutics Announces FDA Clinical Hold on IND Application for DYNE-251 in Duchenne

Dyne Therapeutics recently shared that the FDA issued a clinical hold on their Investigational New Drug (IND) application for the Phase I/II study of their exon skipping therapy utilizing a conjugated monoclonal antibody DYNE-251. For…

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