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Researchers at Solid Biosciences are seeking boys with Duchenne muscular dystrophy (DMD) to participate in a phase 1/2 clinical trial (INSPIRE DUCHENNE) to evaluate the safety and efficacy of the investigational gene therapy (SGT-003). DMD is caused by the loss of the dystrophin protein, which is important for the structure and function of muscle cells. SGT-003…

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Corticosteroids are anti-inflammatory drugs that play an important role in treating some neuromuscular diseases. These medications are the fastest and most efficient way to control inflammation, and they can also reduce pain and suppress overactive immune system responses. According to Dr. Elizabeth McNally, a cardiologist and Director of Northwestern University’s Center for Genetic Medicine in…

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For years, I told the kids we couldn’t get another dog unless something happened to the two we already had. I did not want three dogs! I didn’t have room for that! Yet we now have a new puppy. My life is bursting at the seams most days, given that I’m a hands-on mom who’s…

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My name is Santana Gums and I have LGMD-2b. I live in Arizona and work as a Legal Assistant. Back in February of this year, I attended the Arizona Muscle walk, which just so happened to be my first ever MDA event. Being new to the community, I wasn’t sure what to expect, who would…

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Limited-Edition Violent Gentlemen Hockey Club Tees Benefiting the CureDuchenne. Your purchase makes a difference.Every tee supports CureDuchenne’s mission to improve the lives of everyone affected by Duchenne muscular dystrophy—by accelerating research to find a cure, improving care, and empowering the Duchenne community. Special thanks to Violent Gentleman Hockey Club for standing with us in the…

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Charcot-Marie-Tooth disease (CMT) encompasses a group of inherited disorders that affect movement and sensation in the arms, legs, hands, and feet. Common symptoms include lower leg weakness, foot deformities, reduced sensations, and sometimes impaired fine motor skills. While CMT typically progresses slowly, symptoms may spread over time and lead to secondary complications, such as scoliosis.…

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Researchers at Albany Medical College are seeking parents and caregivers of children with Duchenne muscular dystrophy (DMD) to participate in an observational REDCap survey about their adherence to DMD care guidelines and challenges and priorities in managing their child’s care in the United States. The findings of this survey will help to better understand the experiences…

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Across the U.S., the neuromuscular disease community is preparing to mark Muscular Dystrophy Awareness Month this September, with a series of events and activities aimed at spotlighting muscular dystrophy (MD) and other related conditions. These efforts, organizers say, also seek to raise vital funds to support all individuals in the country who are affected by…

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Researchers at the Center for Health + Technology at the University of Rochester are seeking individuals with non-dystrophic myotonias (NDM) to participate in an observational interview. The findings of the interviews will help to better understand the symptoms experienced by people with NDM. The study This observational study consists of a one-time Zoom interview that will…

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Levels of certain proteins implicated in immune response and the body’s process to stop bleeding differ between Becker muscular dystrophy (BMD) and Duchenne muscular dystrophy (DMD), according to a study Also, blood levels of these proteins correlated with the degree of motor function in BMD participants, but not in those with Duchenne. “If further evaluated…

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