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Pfizer Announces Clinical Hold Lifted for its Mini-Dystrophin Gene Therapy Program

Pfizer has issued a community letter sharing that the FDA has lifted the clinical hold on the company’s Phase 3 ambulatory trial (CIFFREO) for their investigational mini-dystrophin gene therapy product for Duchenne. Pfizer has addressed…

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Solid Biosciences Shares Update on Duchenne Gene Therapy Pipeline Programs

Today Solid Bio shared an update on their gene therapy programs for SGT-001 and SGT-003. SGT-001 is Solid’s AAV9 microdystrophin gene therapy investigational product that is currently being evaluated in boys with Duchenne. SGT-003 is…

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Astellas Gene Therapies Announces plans to discontinue development of Duchenne gene therapy programs

Last night, based on recent preclinical data, Astellas Gene Therapies announced that the research and development of gene therapy programs AT702, AT751 and AT753 for Duchenne Muscular Dystrophy have been terminated. • AT702 was designed…

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Image of young boy participating in Steroid Dosing & Exercise Study at Univ. of Florida

WATCH: Twice Weekly Steroids & Exercise for Duchenne – Study Update & Preliminary Findings (Webinar Recording)

Dr. Tanja Taivassalo joined PPMD for a community webinar last week to present an update on the University of Florida’s Department of Defense funded clinical trial evaluating the potential of low-dose twice weekly steroids and…

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Edgewise-Funded Natural History Trial Of Becker Muscular Dystrophy Now Enrolling

Edgewise Therapeutics has announced the start of an observational trial in participants with Becker muscular dystrophy to understand the disease progression as assessed by functional measures and imaging endpoints. The trial is expected to recruit…

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PPMD Announces First Patients Consented into EHR Study!

PPMD is excited to announce the launch of our new Electronic Health Record (EHR) Study, which will extract data from several of our Certified Duchenne Care Centers across the country, with the first patients now…

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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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