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Gene therapy represents one of the most revolutionary advances in modern medicine, offering hope and healing for previously untreatable genetic disorders. Among these breakthroughs, Zolgensma stands out as a groundbreaking treatment for spinal muscular atrophy (SMA)—a rare but severe neuromuscular disease affecting infants and children.
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Zolgensma (onasemnogene abeparvovec-xioi) is a one-time gene replacement therapy approved by the FDA to treat children under two years with SMA. It delivers a functional copy of the SMN1 gene using an adeno-associated virus vector, restoring the production of the survival motor neuron protein essential for muscle function.
According to the NIH, Zolgensma has transformed SMA treatment, significantly improving motor function and survival rates.
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As explained on Wikipedia, Zolgensma introduces a healthy copy of the SMN1 gene into patients’ motor neurons, enabling them to produce the essential SMN protein. This halts or reverses muscle degeneration, often allowing children with SMA to achieve motor milestones previously deemed impossible.
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If you are searching for where to buy Zolgensma gene therapy in the US, look no further than WorldScientificImpact.org. Here, innovation meets compassion—providing life-saving treatments while fostering hope for the less privileged.
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