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Living with Duchenne muscular dystrophy (DMD) has taught me countless lessons, especially the power of connection, creativity, and celebrating every form of love. Last Saturday, I attended the 11th annual concert of The Purple Symphony with my girlfriend, Amanda, and my new caregiver, Julie. Titled “Perhaps, It’s Love,” the event was held at the Singapore…

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Our recent investment in Entos Pharmaceuticals (https://entospharma.com) aims to explore the potential of their non-viral, redosable Fusogenix PLV platform to deliver full-length dystrophin protein to all muscle groups in a safe, and effective manner with lower cost compared to an adeno-associated virus (AAV) (https://entospharma.com/news-%26-media/f/entos-to-develop-dystrophin-therapy-for-dmd-with-cureduchenne).   Gene therapy is a significant therapeutic option for individuals with…

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A second person with Duchenne muscular dystrophy (DMD) has died of acute liver failure after being treated with the one-time gene therapy Elevidys (delandistrogene moxeparvovec-rokl). A first death was announced in March and both occurred in people with DMD who were no longer able to walk. Following the deaths, the gene therapy’s developer Sarepta is…

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It’s the most wonderful time of the year! The holiday’s season already? No! Summertime! That’s right! The time of year when the days are long and hot, the nights are warm and filled with fireflies, and the atmosphere is prime for active living. And for those in the neuromuscular disease community, summertime is the perfect…

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We are heartbroken to learn of the loss of another member of our community following treatment with ELEVIDYS. According to Sarepta, this represents the second reported case of acute liver failure in a non-ambulatory patient resulting in death following treatment with ELEVIDYS.  Our hearts ache for this family. The second tragic loss following treatment with…

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CureDuchenne is sad to share that another person has passed away due to acute liver failure after treatment with Elevidys, Sarepta’s micro-dystrophin gene therapy.  Our hearts go out to the family and friends of this individual at this difficult time.   While regulators and experts are reviewing the data and looking at possible safety enhancements including additional…

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Ranae Beeker, who is in her late 60s, has found her greatest joy in her roles as a mother to Alethea and Gavin, and as a Gramma to Eilif and Zuzu. Ranae’s journey to diagnosis was not without its challenges, as she underwent three misdiagnoses before finally finding answers at Johns Hopkins. where doctors confirmed…

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AFFINITY DUCHENNE®: New Phase 1/2 Interim Functional Data Watch HERE The post REGENXBIO WEBINAR appeared first on CureDuchenne.

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I’m a mother of seven and a caregiver to the three of my sons who have Duchenne muscular dystrophy (DMD). My oldest daughter, Lexi, 24, lives a few hours away with her husband. I live in rural Nebraska with my husband, Jason, and our six youngest children: Max, 19; Chance, 18; Rowen, 16; Charlie, 14;…

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PPMD is pleased to announce the expansion of its renowned Certified Duchenne Care Center (CDCC) Program with the certification of the Neuromuscular Program at Children’s Hospital of Philadelphia (CHOP). In a milestone for the program, CHOP becomes not only a CDCC, but also the first center to receive PPMD’s newly established Clinical Research Designation—recognizing excellence…

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