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Donnie Demers: The Man Behind the Music
Donnie Demers performing at the Global Green Pre-Oscar Gala (Photo from Donnie’s Facebook page) When looking at the list of accomplishments and accolades that musician and multi-platinum selling song-writer Donnie Demers achieved during his career, it is impossible not to be impressed by his talent and success – but what his family and loved ones…
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Embracing the unfamiliar quiet as my large family grows up
The house is quiet this morning. A dog sleeps next to me on the couch as I contemplate the words I will use to write this column. The news is on, so there is some background noise, and I can faintly hear a video game playing in my son’s room. This level of quiet is…
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FORZETTO Explained: Understanding the Phase 3 Trial of Zeleciment Rostudirsen (DYNE-251) for Duchenne
Dyne Therapeutics: FORZETTO Explained: Understanding the Phase 3 Trial of Zeleciment Rostudirsen (DYNE-251) for Duchenne Watch HERE The post FORZETTO Explained: Understanding the Phase 3 Trial of Zeleciment Rostudirsen (DYNE-251) for Duchenne appeared first on CureDuchenne.
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First Duchenne patient receives novel gene-editing therapy in trial
For the first time, a person with Duchenne muscular dystrophy (DMD) has been given an experimental gene-editing therapy, called PBGENE-DMD, that’s designed to treat the neurodegenerative disease by altering the patient’s own genetic code. Precision Biosciences, the U.S. company developing PBGENE-DMD, announced that dosing had begun in the Phase 1/2 FUNCTION-DMD clinical trial (NCT07429240). The…
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Working hard on staying positive as my FSHD progresses
I stepped back onto the medical treadmill this year to determine if the pain in my right shoulder and elbow, as well as the weakness and stiffness in my right arm and hand, were the result of facioscapulohumeral muscular dystrophy (FSHD). I was hoping it was something else, something that might be treatable. I knew…
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Schooling and muscular dystrophy
Muscular dystrophy (MD) can affect your child’s learning, social life, and physical safety during their school journey. Although your child may face additional challenges while navigating the education system with MD, school accommodations can help them succeed in the classroom. MD is a group of conditions characterized by the progressive weakening and wasting of muscles.…
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Learning disabilities and muscular dystrophy
Navigating school or occupational challenges faced by children with learning disabilities and muscular dystrophy (MD) can feel overwhelming. However, understanding how these two things are linked can help you or the individual you care for connect with the right support. MD is an umbrella term for a class of genetic disorders characterized by progressive muscle…
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Tube feeding and muscular dystrophy
Muscular dystrophy (MD) is a group of inherited muscle diseases that cause progressive muscle weakness and wasting. As the disease progresses, the loss of muscle strength can make all manner of daily tasks challenging, including eating. Some patients find it hard to hold cutlery, chew, and swallow food. One supportive therapy for managing this effect…
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FDA decision on DMD cell therapy pushed to November
The U.S. Food and Drug Administration (FDA) is extending its review of deramiocel, an experimental cell therapy for Duchenne muscular dystrophy (DMD) as it reviews additional data. Capricor Therapeutics, the company developing deramiocel, said it amended its application seeking FDA approval to include new long-term data from the Phase 3 HOPE-3 clinical trial (NCT05126758) and…
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Depression and muscular dystrophy
This story includes discussion of suicide. Support is available if you experience suicidal thoughts, are making plans to self-harm, feel like you or someone you know is unable to stay safe, or are in immediate danger that requires urgent help. If you or someone you know needs urgent mental health support, the following services are…
