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Revised Community-Led Duchenne Guidance Published: Developing Potential Treatments for the Entire Spectrum of Disease
Today PPMD is pleased to share that the revised community-led Duchenne Guidance has been published […]
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Exon-skipping AOC 1044 gets FDA rare pediatric disease status
The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to AOC […]
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Watch: Overview of WVE-N531 and the FORWARD-53 Clinical Trial with Wave Life Sciences (Webinar Recording)
Wave Life Sciences recently joined PPMD for a community webinar to share an update on […]
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I’m tapering off antidepressants and steroids with no regrets
On Feb. 19, I went to see my psychiatrist for the first time in 2024, […]
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SAT-3247 improves muscle function in FSHD mouse model
SAT-3247, an oral therapy candidate for rebuilding muscle tissue, improved muscle function in a mouse […]
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PPMD Announces Pediatric Certified Duchenne Care Center at Penn State Health Children’s Hospital
Today PPMD announced the expansion of our renowned Certified Duchenne Care Center (CDCC) Program with […]
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Sarepta Therapeutics Announces U.S. FDA Acceptance of an Efficacy Supplement to Expand the ELEVIDYS Indication
The FDA will make a decision whether or not to grant full approval of Sarepta’s […]
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The things we don’t talk about as Duchenne caregivers
When three of my sons were diagnosed with Duchenne muscular dystrophy more than 10 years […]
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FDA Accepts Efficacy Supplement to Expand ELEVIDYS Indication
PPMD is excited to learn that the U.S. Food and Drug Administration (FDA) has accepted […]
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How to Identify and Manage Fat Embolism Syndrome (FES) in Fractures
Fat embolism syndrome (FES) is a potentially life-threatening condition that can be triggered by bone […]
