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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 the company’s FORWARD-53 study. Wave provided an overview of initial clinical trial results for WVE-N531 in individuals with Duchenne muscular dystrophy who are amenable to exon 53 skipping, as well as an overview of the potentially registrational FORWARD-53 study. The…
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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, after enduring a rocky start to the new year. When I last saw him in November, we’d decided that I’d consult him again as needed, but I wouldn’t have fixed appointments. The year started for me where 2023 had left…
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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 model of facioscapulohumeral muscular dystrophy (FSHD). Developed by Satellos Bioscience, the therapy is the lead treatment candidate for Duchenne muscular dystrophy (DMD), with the first clinical trial of SAT-3247 for DMD expected to start this year. “We look forward to…
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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 the certification of the clinic at Penn State Health Children’s Hospital in Hershey, Pennsylvania. This designation marks a significant step for the CDCC Program and continues the growth and expansion to bring comprehensive care to all people living with Duchenne…
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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 gene therapy, Elevidys, by June 21,2024. The Efficacy Supplement submitted by Sarepta seeks to broaden the indication for Elevidys to all ages and irrespective of ambulation status. Read the press release: https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-announces-us-fda-acceptance-efficacy The post Sarepta Therapeutics Announces U.S. FDA Acceptance…
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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 ago, I shared everything we experienced on social media and found support and understanding. But as they grow older, weaker, and more dependent on me, I find caregiving harder than I could’ve imagined. I want others to understand our life…
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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 and filed Sarepta Therapeutics’ efficacy supplement to the Biologics License Application (BLA) for ELEVIDYS. The efficacy supplement aims to expand the labeled indication for ELEVIDYS to state: “[ELEVIDYS is indicated for] the treatment of Duchenne muscular dystrophy (DMD) patients with…
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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 fractures. It’s important to know the symptoms and what to do so that you can respond quickly. Check out CureDuchenne’s conversation with Dr. Brenda Wong, a neuromuscular physician from the Duchenne Clinic at the University of Massachusetts, and two parents…
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Even simple questions become complicated with FSHD
Last night, my wife, Wendy, asked me if I’d like to go out to eat. For most people, this request wouldn’t require much thought. It’s a simple yes or no question. However, my facioscapulohumeral muscular dystrophy (FSHD) makes even simple decisions like this one much more complicated. FSHD is a deceptive disease. As I sit…
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CureDuchenne Hosts “Champions in Miami” Event on March 9 to Help Find a Cure for Duchenne Muscular Dystrophy
DJ Irie of the Miami Heat Headlines as Event Brings Together Miami’s Top Business Leaders, Influencers and Philanthropists for a Good Cause Miami, FL, February 15, 2024 – CureDuchenne and the Finazzo family of Miami will host “Champions in Miami” on Saturday, March 9, 2024 at the Kimpton Surfcomber in Miami Beach, bringing together Miami’s top…
