How Does Zolgensma Treatment Work?
Zolgensma, is a gene replacement therapy designed to address the genetic root cause of SMA by delivering a new working copy of the SMN gene into the body. Zolgensma is described as an SMN-enhancing therapy that works by replacing the function of the missing or nonworking SMN1 gene with a new working copy of an SMN gene.
Zolgensma works through an adeno-associated virus vector, often called AAV, which acts like a delivery vehicle to carry the working SMN gene into cells. Once delivered, the new gene helps the body produce survival motor neuron protein, giving motor neurons the support they need to function. In the United States, the FDA lists Zolgensma as an AAV vector-based gene therapy indicated for pediatric patients less than two years old with Spinal Muscular Atrophy caused by bi-allelic mutations in the SMN1 gene. Unlike treatments that require repeated dosing, Zolgensma is given as a one-time intravenous infusion, making it one of the most significant milestones in SMA treatment awareness.
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