March 22, 2024

FDA Grants Approval to DUVYZAT™ (Givinostat)

PPMD is excited to learn that the FDA has granted approval to DUVYZAT™ (givinostat), a histone deacetylase (HDAC) inhibitor indicated for individuals diagnosed with Duchenne muscular dystrophy from six years of age and older. DUVYZAT™…

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March 19, 2024

Italfarmaco Shares Updates Ahead of Givinostat PDUFA Date

Italfarmaco S.p.A. has announced that the full results from the pivotal phase 3 EPIDYS clinical trial of givinostat in ambulant Duchenne patients six years of age and older have been published in The Lancet Neurology….

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March 19, 2024

WATCH: Sarepta Therapeutics – Data update from MOMENTUM study, SRP-5051 (Webinar Recording)

Sarepta Therapeutics recently joined PPMD for a community webinar: Data Update From the MOMENTUM Study of SRP-5051. Sarepta Therapeutics was invited to share data from Part B of MOMENTUM, a Phase 2 Study of SRP-5051…

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March 19, 2024

PTC Therapeutics Announces Plans to Resubmit Translarna™ NDA to FDA

PTC Therapeutics, Inc. has announced that based on recent feedback from the FDA, the company plans to re-submit a New Drug Application (NDA) for Translarna™ (ataluren), a dystrophin restoration therapy via stop-codon readthrough for the…

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March 15, 2024

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

NS Pharma recently joined PPMD for a community webinar to share an overview of two of the company’s new exon skipping trials: Brogidirsen (exon 44) and NS-050/NCNP-03 (exon 50). Vamshi Rao, MD, discussed these two…

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March 15, 2024

The Duchenne Registry Has a New Gene Therapy Survey!

Open The Duchenne Registry app and you will notice a new survey on Gene Therapy. If you do not see the Gene Therapy survey listed under the Activities section, please update your app. We recently…

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March 13, 2024

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

PPMD is pleased to learn of the U.S. commercial launch of AGAMREE® (vamorolone) oral suspension 40 mg/mL for the treatment of Duchenne in patients aged two years and older. According to Catalyst Pharmaceuticals, Inc., following…

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March 5, 2024

REGENXBIO Shares New Positive Initial Efficacy Data From AFFINITY DUCHENNE Trial

REGENXBIO Inc. has shared an update on the Phase I/II AFFINITY DUCHENNE® trial of RGX-202 in patients with Duchenne ages 4 to 11 years old, including RGX-202 microdystrophin expression from dose level 2. RGX-202 is…

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February 23, 2024

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

Wave Life Sciences recently joined PPMD for a community webinar to share an update on the company’s FORWARD-53 study. Wave provided an overview of initial clinical trial results for WVE-N531 in individuals with Duchenne muscular…

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February 23, 2024

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

Today PPMD is pleased to share that the revised community-led Duchenne Guidance has been published in the Journal of Neuromuscular Diseases! This Community Draft Guidance was submitted to the FDA at the end of 2022….

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