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Duvyzat now available to DMD patients 6 and older in US
Duvyzat (givinostat), a newly approved treatment for Duchenne muscular dystrophy (DMD), is now available in […]
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PepGen Announces Positive Data from Phase 2 Clinical Trial of PGN-EDO51
PepGen Inc. has shared positive clinical data from the first dose cohort (5 mg/kg) of […]
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Finding Belonging and Fostering Community
While no one asks to be part of this community, and entering the world of […]
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Pfizer Discontinues Development of Investigational Mini-Dystrophin Gene Therapy
PPMD is disappointed to learn that Pfizer Inc. has officially discontinued development of the company’s […]
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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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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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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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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 […]
