User:Mr. Ibrahem/Onasemnogene abeparvovec

Onasemnogene abeparvovec, sold under the brand name Zolgensma, is a medication used to treat certain types of spinal muscular atrophy (SMA). It is used as a one-time gradual injection into a vein with at least 2 months of corticosteroids. It is used in children less than two years old.

Common side effects include vomiting and increased liver enzymes. Serious side effects may include liver failure and low platelets. There is also evidence of damage to the heart; however, this is of unclear significance. Onasemnogene abeparvovec is a type of gene therapy and works by providing a new copy of the gene that makes the human SMN protein.

Onasemnogene abeparvovec was approved for medical use in the United States in 2019. In 2020 it was approved in Japan, Brazil, Europe, and Canada. At launch, it cost about US$ 2.125 million per treatment in the United States, making it one of the most expensive medications.