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Your Citizen Science At Work

By sharing your anonymous data through The Duchenne Registry, you are strengthening the power of a 15-year-old network of patient-powered data that is used to improve care for people living with Duchenne and increase our…

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Clinical Trials & Outcome Measures in Duchenne

Clinical trials and research studies are vitally important to improving health and quality of life for people with Duchenne and Becker. Research is always advancing and evolving, and outcome measures fall into this category, as…

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REGENXBIO Announces Positive Data From AFFINITY DUCHENNE® Trial of RGX-202

REGENXBIO Inc. has shared new, positive interim safety and efficacy data in the Phase I/II AFFINITY DUCHENNE® trial of RGX-202 in participants with Duchenne ages 1 to 11 years old. RGX-202 utilizes a novel adeno-associated…

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PepGen Announces Positive Data from Phase 2 Clinical Trial of PGN-EDO51

PepGen Inc. has shared positive clinical data from the first dose cohort (5 mg/kg) of PGN-EDO51, the company’s lead investigational candidate for patients with Duchenne whose mutations are amenable to exon 51-skipping. PGN-EDO51 is an…

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Finding Belonging and Fostering Community

While no one asks to be part of this community, and entering the world of Duchenne and Becker can be overwhelming, you are not alone. During his keynote session at PPMD’s 30th Annual Conference, Brandon…

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Pfizer Discontinues Development of Investigational Mini-Dystrophin Gene Therapy

PPMD is disappointed to learn that Pfizer Inc. has officially discontinued development of the company’s investigational mini-dystrophin gene therapy, fordadistrogene movaparvovec. This announcement follows Pfizer’s June 2024 update on the Phase 3 CIFFREO study evaluating…

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Response to the July 29th STAT Article

A brief history: During and since 2016, there has been frustration in this community regarding drug development. Fast forward: PPMD strives for both accuracy and transparency in all information we release. We made the decision…

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Making it Work: Practical Tips for Enhancing Quality of Life

It’s important to ensure that even within the context of Duchenne/Becker, kids are doing things that kids should be doing, adults are doing things that adults should be doing, and families can lead the lives…

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ITF Therapeutics Announces U.S. Commercial Availability of DUVYZAT™ (givinostat) for Treatment of Duchenne

PPMD is pleased to learn that DUVYZAT™ (givinostat) is now commercially available in the United States. DUVYZAT™ is a histone deacetylase (HDAC) inhibitor indicated for individuals diagnosed with Duchenne from six years of age and…

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Navigating the Approved Therapy Landscape

The approved therapy landscape has changed drastically in Duchenne, especially over the last year. With multiple newly approved therapies comes a lot of questions about how to navigate these drugs: when they are prescribed; who…

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