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PTC Therapeutics Announces Plans to Resubmit Translarna™ NDA to FDA

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WATCH: NS Pharma – Overview of New Exon Skipping Trials: Brogidirsen and NS-050/NCNP-03 (Webinar Recording)

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The Duchenne Registry Has a New Gene Therapy Survey!

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Join Us in Orlando for PPMD’s 30th Annual Conference

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Catalyst Pharmaceuticals Announces AGAMREE® Now Commercially Available in the U.S. for the Treatment of Duchenne

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REGENXBIO Shares New Positive Initial Efficacy Data From AFFINITY DUCHENNE Trial

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PPMD Hosts Town Hall on Clinical Trials During 2024 Advocacy Conference

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PPMD Celebrates 30th Anniversary and Three Decades of Progress with Annual Advocacy Conference

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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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