Zolgensma
Zolgensma is a gene therapy treatment intended for people with a rare group of genetic conditions known as spinal muscular atrophy. The treatment provides patients with a healthy copy of the SMN gene, allowing their cells to produce SMN protein, which is vital to the health of motor neurons.
The single-dose treatment costs an astonishing $2.1 million in the U.S. as of mid 2022, which made it the most expensive medication until the approval of Zynteglo in August 2022. It isn’t free of severe side-effects either. The drug’s maker, Novartis, reported in August that two patients in Russia and Kazakhstan died of liver failure weeks after starting therapy. However, the drug’s benefits still outweigh its risks, the company argued.
