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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…
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Edgewise Therapeutics’ EDG-5506 on FDA fast track for Duchenne MD
EDG-5506, an oral treatment being developed by Edgewise Therapeutics, has been granted fast track designation for Duchenne muscular dystrophy (DMD) by the U.S. Food and Drug Administration (FDA). The agency gives this designation to experimental therapies that have the potential to fill unmet medical needs, with the aim of getting much-needed medications to market faster.…
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Cognitive abilities stable over time in Becker MD boys, study finds
Cognitive function in boys with Becker muscular dystrophy (BMD) appear to be stable over time, although these children may struggle with working memory and executive function, a small study reports. Based on its findings, the researchers are calling for boys with BMD to have access to routine cognitive and psychiatric monitoring so that, if issues…
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WATCH: PTC Therapeutics — EMFLAZA® Landscape in 2024 (Webinar Recording)
PPMD was recently joined by the team at PTC Therapeutics to discuss upcoming changes and continuing community support around EMFLAZA (deflazacort). EMFLAZA came on the U.S. market in 2017 as the first FDA-approved steroid specifically indicated for the treatment of Duchenne. Since then, it has been available exclusively through PTC. However, earlier this month, patents…
