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WATCH: Advancing DYNE-251 – An Investigational Medicine for Duchenne to Deliver for Patients (Webinar Recording)
Dyne Therapeutics recently joined PPMD for a presentation of clinical data from the DELIVER Trial of DYNE-251 in individuals living with Duchenne muscular dystrophy who are amenable to exon 51 skipping, first released on May 20th, 2024. Dyne’s Chief Medical Affairs Officer and VP of Global Patient Advocacy shared insights on how these data demonstrated…
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CureDuchenne Expands Global Initiatives and Spreads Awareness for World Duchenne Day 2024
International-Outreach-and-World-Duchenne-Day-2024-FINALDownload Newport Beach, CA – August 15, 2024 — CureDuchenne, a global leader in research, patient care and innovation for improving and extending the lives of those with Duchenne muscular dystrophy, proudly recognizes World Duchenne Day, celebrated on September 7, by launching new global initiatives and amplifying awareness of the disease. Duchenne muscular dystrophy is…
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Confronting the financial burdens of DMD with a heap of faith
I have seven children, and three of them are living with Duchenne muscular dystrophy (DMD): Max, 18, Rowen, 15, and Charlie, 13. If you’re a caregiver or a parent, I’m sure you’ll understand how tired I am. I won’t peruse my past columns, but I’m sure I’ve written about how worn out I get. So…
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Why having my own ‘escape hatch’ is key to my happiness
Roughly four decades into living with limb-girdle muscular dystrophy, I’ve developed numerous ways to thrive. Like many others, I believe in the power of a positive attitude. I try my best to project a positive outlook when communicating with others, whether it’s face to face or by using words in a column that project out…
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Del-zota increases dystrophin in DMD patients, new trial data show
Avidity Biosciences‘ delpacibart zotadirsen, or del-zota for short — formerly called AOC 1044 — was found to safely increase levels of dystrophin production to 25% of normal in people with Duchenne muscular dystrophy (DMD), according to new data from the EXPLORE44 trial. In the Phase 1/2 clinical trial (NCT05670730), which enrolled DMD patients with mutations…
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Ladies Luncheon Newport Beach to Debut at Shady Canyon Golf Club on October 15 Benefiting CureDuchenne Inaugural Event Features Fashion, Food, and Fun to Raise Funds for Cutting-Edge Duchenne Muscular Dystrophy Research
NEWPORT BEACH, California (August 13, 2024) – CureDuchenne, a global nonprofit dedicated to funding and finding a cure for Duchenne muscular dystrophy, is excited to announce the inaugural Ladies Luncheon Newport Beach at Shady Canyon Golf Club on Tuesday, October 15, 2024. The event, which promises an elegant afternoon of fashion, food, and fun, will…
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Adjusting my definition of what it means to be a man
Kevin Schaefer, the associate director of community content at Bionews, the parent company of this website, recently shared a thought-provoking article from Psychology Today titled “How Chronic Illness and Masculinity Intersect.” It has me thinking about what kind of man I am and want to be. The article notes that “changes caused by chronic illness…
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SAT-3247 for DMD wins FDA rare pediatric disease designation
The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to SAT-3247, an oral treatment candidate for Duchenne muscular dystrophy (DMD) that ultimately aims to slow the progression of the genetic condition. The FDA awards this status to investigational medicines that are designed to treat serious, rare diseases that mainly affect children.…
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Finding Support to Manage Behaviors in Duchenne and Becker
Research has shown that dystrophin is usually present in the brain as well as in the muscles. Scientists believe that when dystrophin is missing or partially functional, like in Duchenne and Becker, brain neurons may not function as well as they should. This can lead to learning and behavior challenges for some children. While not…
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Solid Biosciences has begun dosing patients in their clinical study of their next-generation gene therapy, SGT-003 for those living with Duchenne.
Letter to the Duchenne Community – INSPIRE DUCHENNE update Dear Duchenne Community, We are writing to share an important update on INSPIRE DUCHENNE, our Phase 1/2 clinical trial of SGT-003 for the treatment of patients living with Duchenne. At Solid Biosciences, our mission is driven by the needs and hopes of the Duchenne community, and we…
