New Gene Therapy Transforms SMA (Spinal Muscular Atrophy)
URMC has been tapped as one of the first institutions in the U.S. to offer a new gene replacement therapy to treat spinal muscular atrophy (SMA). Until a few years ago, an SMA diagnosis was tantamount to a death sentence. Learn how this cutting-edge therapy is transforming the lives of patients and their families.
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Charlie and Rupert on their journey with Spinal Muscular Atrophy and Zolgensma Gene Therapy
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Reagan Goforth, Spinal Muscular Atrophy | Stem Cell Treatment Testimonial
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Cut and Paste: Treating Spinal Muscular Atrophy with Nusinersen
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Duchenne Muscular Dystrophy
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Medical Stories - Neurofibromatosis Type 1: Aubrie's Story
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Baby Among the First to Receive Gene Therapy for Spinal Muscular Atrophy After FDA Approval
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Spinal Muscular Atrophy 2019: SMA Pathophysiology, Diagnosis and Clinical Presentations
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Ruzgar's story with Spinal Muscular Atrophy (SMA)
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Parents race to save toddler’s life after rare genetic disease diagnosis | Nightline
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Spinal Muscular Atrophy 2019: Approved Treatment and Upcoming Therapeutic Strategies
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April 2022 Cure SMA and Novartis Gene Therapies Clinical Trial Update Webinar
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Finding a cure for spinal muscular atrophy (SMA)
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Update on Zolgensma in the UK - Feb 2024
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SMA and the drug that fights it, explained
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The Lethal Drug Trial That Turned Men Into Elephants
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Gene Therapy for SMA Type 1: Evelyn's Story
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Genetics of SMA
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Living with Juvenile Huntington's Disease
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Chase’s Story | UR Medicine
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