Zolgensma: The Game Changing Gene Therapy for SMA Families
Zolgensma, is considered a game-changing gene therapy because it is designed to address the genetic root cause of SMA by delivering a working copy of the SMN gene into the body. In the United States, Zolgensma is an FDA-approved gene therapy for pediatric patients less than 2 years old with SMA.
Zolgensma works by using an adeno-associated virus vector, often called AAV9, to carry a working SMN gene into cells so the body can begin producing survival motor neuron protein. This gene therapy helps replace the function of the missing or nonworking SMN1 gene with a new working copy of an SMN gene. Early treatment conversations can become urgent when motor neurons are vulnerable, and muscle weakness can progress quickly. Unlike some SMA treatments that require ongoing treatment, Zolgensma is given as a one-time intravenous infusion, making it one of the most significant milestones in the history of Spinal Muscular Atrophy treatment.
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