Zolgensma (Onasemnogene Abeparvovec)
Zolgensma (Onasemnogene Abeparvovec)
Zolgensma – how it works, is given, and safety considerations – FAQs
Onasemnogene abeparvovec (Zolgensma™) uses harmless, genetically-engineered viruses to increase SMN protein levels. A single injection of Zolgensma into the blood is designed to address the genetic root cause of SMA by replacing the faulty or missing SMN1 gene.
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Zolgensma Trials and Their Results
Intravenous Zolgensma has been tested in human clinical trials, with children up to the age of 2 years, since 2014. It has been consistently shown to have a well-understood safety profile and to be well-tolerated in these trials.
In many children who have SMA, when compared to no treatment, zolgensma has been shown to increase the availability of SMN protein, leading to clinically meaningful improvements in muscle function. Several trials of IV – delivered Zolgensma are currently ongoing with a variety of children, including those who are older and heavier than in the initial trials
A trial investigating intrathecal administration of the treatment with older heavier children resumed in August 2021.
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