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Stem Cell Treatment for SMA - Spinal Muscular Atrophy
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What Is SMA? | ZOLGENSMA®
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zolgensma.com
321K views · 2.4K reactions | Today, the Food and Drug Administration approved a new gene therapy for Spinal Muscular Atrophy. SMA Type 1 is the number one genetic killer of children 2 and under. This first-time gene therapy, developed here at Nationwide Children's, is allowing kids like Evelyn to have a bright future. Watch her incredible story. | Nationwide Children's Hospital | Facebook
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denver7.com
3.1K views · 148 reactions | Itvisma (onasemnogene abeparvovec-brve) is a gene therapy that works by delivering a functional copy of the SMN1 gene directly to motor neurons, helping the body restore SMN protein production and slow or halt SMA progression. In clinical trials, it was shown to improve motor function, provide rapid therapeutic effect, and expand treatment options for children and adults age 2 and older living with SMA. | Allie Wheelz | Facebook
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3.1K views · 148 reactions | Itvisma (onasemnogene abeparvovec-brv…
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