Gene Therapy Process | ZOLGENSMA®
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Gene Therapy Process | ZOLGENSMA®
Discover how a single ZOLGENSMA® infusion can stop the progression of SMA. See Safety and Full Prescribing Information. For US audiences only.
Jun 24, 2019
Onasemnogene abeparvovec Uses
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
1:43
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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1.7K views1 month ago
Novartis Pharma AG: Novartis intrathecal onasemnogene abeparvovec Phase III study meets primary endpoint in children and young adults with SMA
Novartis Pharma AG: Novartis intrathecal onasemnogene abeparvovec Phase III study meets primary endpoint in children and young adults with SMA
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