Zolgensma

Zolgensma is a gene therapy treatment designed for spinal muscular atrophy (SMA), a severe genetic disorder that affects muscle control and movement in children. SMA is caused by a missing or defective gene that leads to the loss of motor neurons in the spinal cord. Zolgensma works by delivering a healthy copy of this gene directly to the patient's cells, enabling them to produce the missing protein necessary for motor neuron survival. This treatment can significantly improve motor function and overall quality of life, especially when administered early in life.