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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 […]
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Agamree, next-gen steroid, now available for DMD patients in US
Agamree (vamorolone) is now available for Duchenne muscular dystrophy (DMD) patients, ages 2 and older, […]
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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 […]
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PTC Therapeutics Provides Key Regulatory Updates
PTC Therapeutics announced that, based on recent feedback from the FDA, by mid-year they plan […]
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7 tips for traveling alone with my 3 sons who have Duchenne
I am traveling this week with my three sons who have Duchenne muscular dystrophy (DMD): […]
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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 […]
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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. […]
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MDA 2024: Dyne trials raising doses after early positive results
DYNE-251 and DYNE-101, Dyne Therapeutics’ investigational therapies for forms of muscular dystrophy, appear safe and […]
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MDA 2024: Limb, heart function preserved with DMD cell therapy
The investigational cell therapy CAP-1002 has continued to slow declines in upper limb and heart […]
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Ask Me Anything: Traveling with Duchenne with Marissa Penrod, Adam Malone and Alan Chaulet
This pre-recorded webinar delves into the world of travel with special guests, Marissa Penrod and […]
