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PPMD Awards $250,000 to Support Clinical Research Network for Duchenne Babies Identified by Newborn Screening

PPMD is excited to announce a $250,000 award to Bo Hoon Lee, MD, from the University of Rochester to support the development of a clinical research network for Duchenne babies identified through newborn screening efforts…

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PPMD Celebrates 30th Anniversary with Launch of PPMD Together Meeting Series in Cincinnati, Ohio

PPMD is excited to mark its 30th anniversary at the launch of the PPMD Together meeting series, taking place April 19-20, 2024, in Cincinnati, Ohio. Founded three decades ago by a small group of passionate parents and grandparents…

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

Celebrate three decades of progress, community, and resilience at PPMD’s 30th Annual Conference, June 27–29, 2024 in Orlando, Florida! Back in 1994, our very first Conference was held in Orlando, and now, 30 years later,…

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