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Fracture Management for Duchenne Muscular Dystrophy: Be Prepared with R.A.R.E
Dealing with a fracture is a frightening experience for anyone, especially someone living with Duchenne muscular dystrophy, who has weaker bones and is more prone to falling. Often, you will have to work with emergency room staff who aren’t as familiar with Duchenne and don’t know the signs of fatty embolism syndrome (FES). Then you,…
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A recent fall has me wondering: Should I use a walker?
I had an incident about a month ago that I’ve been thinking about ever since. It’s left me wondering if it’s time for me to begin using a walker, something I tried years ago without much success. The Sunday after my wife, Wendy, had surgery recently, a friend brought me home from church. He dropped…
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Exondys 51 extends survival in DMD, long-term study shows
Up to eight years of Exondys 51 treatment (eteplirsen or AVI-4658) extended survival in Duchenne muscular dystrophy (DMD) patients over a wide range of ages, a long-term study concluded. Patients treated for longer periods had the lowest risk of death. The study, “Survival among patients receiving eteplirsen for up to 8 years for the treatment…
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60+ Experts Unite to Author Consensus Statement Championing Duchenne Newborn Screening
Over 60 leading experts in genetics, pediatrics, neurology, and public health advocates have authored a consensus statement in support of implementing Duchenne newborn screening. Key points of the consensus statement include: Timely Intervention: Early screening allows for prompt intervention and therapeutic strategies, slowing disease progression. Reduced Diagnostic Delays: Screening eliminates diagnostic delays, reducing avoidable costs…
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Reflecting on the beauty in life with Duchenne muscular dystrophy
I have three sons with Duchenne muscular dystrophy (DMD): Max, 18, Rowen, 15, and Charlie, 13. While caregiving and parenting three young men with Duchenne is full of the challenges and heartaches I often share in this column, they do not outweigh the beautiful moments. Joy and suffering coexist with Duchenne, much as they do…
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Revolutionizing Duchenne Care: A Year of Milestones at The CureDuchenne Clinic
This past year the CureDuchenne Clinic at the Neurology & Neuromuscular Care Center, led by Dr. Diana Castro in Denton, TX, has been marked by unparalleled advancements, notably with the administration of the first gene therapy for Duchenne muscular dystrophy. The CureDuchenne Clinic has shown the power of care — not just medical care —…
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CureDuchenne Webinar with italfarmaco | Community Update with ITF Therapeutics: Introducing DUVYZAT™ (givinostat)
Please join us for a session with ITF Therapeutics. Meet members of the ITF Therapeutics team to learn more about their organization and next steps to support the availability of DUVYZAT. Recorded Tuesday, April 2, 2024 Watch Recording The post CureDuchenne Webinar with italfarmaco | Community Update with ITF Therapeutics: Introducing DUVYZAT™ (givinostat) appeared first…
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Gene therapy SGT-003 wins FDA’s rare pediatric disease designation
The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease status to SGT-003, a next-generation gene therapy candidate for Duchenne muscular dystrophy (DMD) developed by Solid Biosciences. The designation is given to therapies with the potential to prevent or treat rare diseases that primarily affect children and adolescents. If it’s approved, Solid may qualify…
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Rollout expected by fall in US for new DMD therapy Duvyzat
ITF Therapeutics — which will be responsible for marketing Duvyzat (givinostat), Italfarmaco’s newly approved Duchenne muscular dystrophy (DMD) therapy, in the U.S — expects the oral medication to be available to eligible adults and children by this fall. For ITF, the U.S.-based rare disease division of Italfarmaco, last month’s approval of Duvyzat by the U.S. Food…
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When life with Duchenne is hard, I remember the value of friendship
During the month of March, I took a break from writing my column because I was experiencing withdrawal from recently tapering off an antidepressant and a steroid medication, which took a toll on my mental and physical health. The withdrawal symptoms aggravated my comorbid attention-deficit/hyperactivity disorder and obsessive-compulsive disorder and caused fatigue and insomnia. They…
