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Air travel, even in the best circumstances, can be a little stressful. Flying with a power wheelchair or other mobility or medical equipment adds complications and hurdles to getting on an airplane. Lyza Weisman loves to travel to new places. But for many people with neuromuscular diseases, the benefits of travel are worth the challenges.…

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Edgewise Therapeutics, which received early funding from CureDuchenne, has reported positive results in its sevasemten program for Becker and Duchenne muscular dystrophies. New open-label data in Becker from the MESA trial demonstrated sustained disease stabilization up to three years in participants, reinforcing prior clinical findings from the ARCH and CANYON trials. Edgewise also reported encouraging topline data from…

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Edgewise Therapeutics, Inc., has announced positive results in the company’s sevasemten program for both Becker and Duchenne. These data include results from: MESA, an open label extension trial of participants with Becker who were previously enrolled in the ARCH trial, or completed the CANYON, GRAND CANYON, or DUNE trials  Phase 2 Duchenne trials, LYNX and…

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I’m an early riser. As I mentioned in a past column, I like to wake up early, get dressed, make coffee, and start the day with some quiet prayer and devotion time. However, I’m also a very sound sleeper, with some help from melatonin, sleep medication, and a comfy mattress. From the time I fall…

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The U.S. Food and Drug Administration (FDA) is due to convene an advisory committee meeting next month to review the data on deramiocel, an experimental cell therapy up for approval to treat heart disease in people with Duchenne muscular dystrophy (DMD). The FDA has informed Capricor Therapeutics the meeting should happen July 30, though Capricor…

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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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