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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 […]
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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 […]
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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, […]
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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 […]
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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 […]
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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, […]
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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 […]
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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 […]
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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 […]
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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 […]
