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Duchenne Siblings Study Published: Assessing Early Diagnosis from the Perspective of Parents with Multiple Children Diagnosed with Duchenne

For more than a decade, PPMD has been at the forefront of advocating for Duchenne newborn screening. In 2023, we expanded our ongoing work in the space by conducting a parent survey to build evidence…

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60+ Experts Unite to Author Consensus Statement Championing Duchenne Newborn Screening

Over 60 leading experts in genetics, pediatrics, neurology, and public health advocates have authored a consensus statement in support of implementing Duchenne newborn screening. Key points of the consensus statement include: Timely Intervention: Early screening…

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WATCH: Community Update with ITF Therapeutics – Introducing DUVYZAT™ (givinostat) (Webinar Recording)

ITF Therapeutics recently joined PPMD for a webinar to provide the Duchenne community with an opportunity to meet members of the ITF Therapeutics team to learn more about their organization and next steps to support…

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ITF Therapeutics Shares Update on DUVYZAT™ Following FDA Approval

Following the FDA’s approval of DUVYZAT™ (givinostat) on March 21, 2024, ITF Therapeutics, LLC, the company responsible for producing and marketing DUVYZAT™ in the United States, today shared an update on the drug and related…

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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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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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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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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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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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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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