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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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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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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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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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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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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…

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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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PPMD is excited to announce that the organization has provided $500,000 in funding to Kinea Bio, Inc. (Kinea) through PPMD Venture Pathways, the organization’s venture philanthropy program which provides industry funding to accelerate therapeutic development. This funding commitment aims to address current limitations in gene therapy delivery and will advance the development of the company’s…

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With projects spanning gene therapy research, patient advocacy, and air travel safety, seven U.S. organizations will receive $140,000 in overall funding from the Muscular Dystrophy Associations (MDA) Advocacy Collaboration Grant Program. The program, which opened in 2022, seeks to support and enhance key public policy and advocacy initiatives through grassroot efforts. The overarching goal is to…

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Pulling into a parking spot at my oldest son’s high school, I hit a parked car with my van. Thankfully, there was no damage to either vehicle, and the car’s owner, sitting in the front seat when I hit it, was amiable and understanding. I could safely unload my son Max, but I didn’t make…

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