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Pfizer Duchenne Program Discontinuation
Pfizer Duchenne Program Discontinuation: Pfizer has officially decided to discontinue their microdystrophin gene therapy program […]
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Response to the July 29th STAT Article
A brief history: During and since 2016, there has been frustration in this community regarding […]
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New gene therapy approach shows promise for Duchenne muscular dystrophy
Researchers have made a significant breakthrough in developing a new gene therapy approach that restores […]
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As a caregiver for my sons with DMD, how do I know what is right?
Wrestling videos were the soundtrack to my morning earlier this week. I was staring at […]
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New study may help more DMD patients be included in clinical trials
Most children with Duchenne muscular dystrophy (DMD) start experiencing notable issues with arm function and […]
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Making it Work: Practical Tips for Enhancing Quality of Life
It’s important to ensure that even within the context of Duchenne/Becker, kids are doing things […]
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ITF Therapeutics LLC Announces U.S. Commercial Availability of DUVYZAT™ (givinostat) for Treatment of Patients With Duchenne Muscular Dystrophy
First nonsteroidal treatment approved for patients six years of age and older with DMD regardless […]
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ITF Therapeutics Announces U.S. Commercial Availability of DUVYZAT™ (givinostat) for Treatment of Duchenne
PPMD is pleased to learn that DUVYZAT (givinostat) is now commercially available in the United […]
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FDA OKs clinical trial of DMD stem cell therapy MyoPAXon
The U.S. Food and Drug Administration (FDA) has given the green light for Myogenica to […]
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I wish more facilities considered accessibility and privacy
A few years ago, my wife, Wendy, and I took a mini-vacation to Philadelphia with […]
