Description
Zolgensma is a prescription gene therapy used to treat spinal muscular atrophy (SMA) — a rare and serious genetic disorder that affects the muscles used for movement, caused by a missing or defective SMN1 gene. It contains onasemnogene abeparvovec-xioi, a specially designed virus that delivers a healthy copy of the SMN1 gene into motor neuron cells, enabling the body to produce the survival motor neuron (SMN) protein essential for muscle function.
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