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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…
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Having the support of friends, old and new, makes all the difference
I received an overwhelming response to my last column, in which I grappled with feelings of uselessness when my facioscapulohumeral muscular dystrophy (FSHD) prevented me from helping in the way I would have liked to do. In this case, I was sitting at home while my wife, Wendy, was undergoing surgery. Many of my readers,…
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Managing motherhood and caregiving: Where is the beauty?
My mind is spinning like the wheels on my son’s power wheelchair in the freshly fallen snow. You did not read that wrong, and it’s not a hypothetical. Here in Nebraska, it snowed this last week of March. It feels like a second winter. As if the spring snow isn’t enough, life is going full…
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Trial will test gene therapy for limb-girdle muscular dystrophy type
Regulators in France and Italy have given Atamyo Therapeutics the green light to launch a clinical trial testing ATA-200, an investigational gene therapy for children with a type of limb-girdle muscular dystrophy called LGMD2C or LGMDR5. The multicenter, dose-escalation Phase 1b study (NCT05973630) — which will assess the safety, efficacy, and pharmacological and immunological properties…
