Zolgensma
The Food and Drug Administration approved the first gene therapy for a type of spinal muscular atrophy that affects children. The disease is caused by a defect in a gene that makes a protein(SMN) necessary for the survival of motor neurons. Zolgensma uses a re-engineered virus to deliver a functional copy of the defective gene so that SMN protein can be produced. Other than Botulinum toxin, Zolgensma is the most expensive drug costing as much as $2.125 million over 5 years, or an annualized cost of $425,000 per year for five years.
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Contributors: Prab R. Tumpati, MD
