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
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People living with spinal muscular atrophy (SMA) are born with a genetic mutation in their SMN1 gene, meaning there is a deficiency in the protein that keeps motor neurons healthy. Without this protein, motor neurons can't send signals properly, causing muscles to start to atrophy. Learn more about the science behind this #raredisease. https://bit.ly/3axDMPZ | Genentech
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