June 2025

Capricor Therapeutics, which received early funding from CureDuchenne, has indicated that the FDA has decided that an Advisory Committee meeting is not warranted at this time.  Capricor’s Biologics License Application (BLA) remains under Priority Review with PDUFA target action date of August 31, 2025 Read the full press release HERE The post Regulatory Update on…

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Capricor Therapeutics has shared a regulatory update related to its Biologics License Application (BLA) for Deramiocel, the company’s novel cell therapy for the treatment of Duchenne-associated cardiomyopathy. As part of the FDA’s ongoing review, Capricor has been informed that an Advisory Committee (AdComm) meeting is not indicated at this time, and the previously scheduled meeting…

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Jennifer Lane is a wife and mother of three grown children and lives in Alabama. She is involved in church ministry and is an advocate and Ambassador for the MDA. She was diagnosed with LGMD 1C in 2022, after 13 years of searching for a diagnosis. Jennifer is a published artist, loves going to concerts,…

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Genetic testing is an important part of diagnosing and managing neuromuscular diseases, including muscular dystrophies, spinal muscular atrophy (SMA), amyotrophic lateral sclerosis (ALS), and Pompe disease. Genetic counselor Jennifer Roggenbuck “Since many neuromuscular diseases have a genetic basis, genetic testing may be the quickest and least invasive way to get a diagnosis,” says Jennifer Roggenbuck,…

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Here in the Midwest, June brings warmer temperatures. I live in Nebraska, where warmer weather is welcome after bitterly cold winters and brisk, windy springs. It’s also necessary for the crops growing in the large fields that line most of the highways. I wait all spring for the temperature to rise above 90 degrees. I…

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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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