Zolgensma: World’s most expensive drug of choice; Survival of Motor Function for the Treatment of Spinal Muscular Atrophy Type 1
Keywords:SMA, SMN1, SMN2, AAV9, capsid, viral DNA
Onasemnogene abeparvovec, sold under the brand name Zolgensma, is a gene therapy medication used to treat spinal muscular atrophy (SMA). It is used as a one-time infusion into a vein. Onasemnogene abeparvovec works by providing a new copy of the gene that makes the human SMN protein. The treatment must be accompanied by a course of corticosteroids of at least two months. Common side effects include vomiting and increased liver enzymes. Onasemnogene abeparvovec was first approved for medical use in the United States in 2019 as a treatment for children less than two years old. It was later approved in other jurisdictions with similar scope. The approval scope in certain jurisdictions, including the European Union and Canada, is somewhat different. Zolgensma TM uses harmless, genetically engineered viruses to increase SMN protein levels. Once the virus is introduced into a person, it is able to travel around the body and get to a variety of different cells to help restore some of the SMN protein that is missing in SMA. Clinical studies have shown Zolgensma to be effective for treating SMA caused by genetic mutations in the SMN1 gene. The drug works by replacing an abnormal SMN1 gene with a normal SMN1 gene. However, Zolgensma won't reverse any effects of SMA that happened before your child received the drug.
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