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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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PPMD’s 2024 Advocacy Conference Brings Together Community to Support Progress in Fighting for Every Future

Earlier this month, more than 120 PPMD advocates – people with Duchenne and Becker, parents, siblings, grandparents, and other family members and friends – from 26 different states gathered in Washington, D.C. to urge legislators…

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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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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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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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PPMD Hosts Town Hall on Clinical Trials During 2024 Advocacy Conference

The PPMD Town Hall on Clinical Trials, taking place on Monday, March 4th during PPMD’s 2024 Advocacy Conference in Washington, D.C., aims to provide a platform for families affected by Duchenne and Becker to share…

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