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Blog

Breaking News: Rare Disease Day Congressional Hearing to Address the BENEFIT Act

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Watch: Overview of WVE-N531 and the FORWARD-53 Clinical Trial with Wave Life Sciences (Webinar Recording)

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Revised Community-Led Duchenne Guidance Published: Developing Potential Treatments for the Entire Spectrum of Disease

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PPMD Announces Pediatric Certified Duchenne Care Center at Penn State Health Children’s Hospital

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FDA Accepts Efficacy Supplement to Expand ELEVIDYS Indication

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WATCH: PTC Therapeutics — EMFLAZA® Landscape in 2024 (Webinar Recording)

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PPMD Provides $500,000 in Funding to Kinea Bio Through PPMD Venture Pathways Program to Support Next-Gen Midi-Dystrophin Gene Therapy Development

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Update From PPMD’s 2024 PPMD Duchenne Healthcare Professionals Summit

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Watch: Dyne Therapeutics — Advancing DYNE-251, an Investigational Medicine for DMD (Webinar Recording)

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REGENXBIO Announces Completion of Enrollment in Cohort 2 and Additional Positive Interim Data in AFFINITY DUCHENNE® Trial

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