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Onasemnogene abeparvovec

The meaning of «onasemnogene abeparvovec»

Onasemnogene abeparvovec, sold under the brand name Zolgensma, is a gene therapy medication used to treat spinal muscular atrophy (SMA).[3] It was approved for children less than two years old in 2019.[5][3] It is used as a one-time injection into a vein with at least two months of corticosteroids.[3]

Common side effects include vomiting and increased liver enzymes.[3][4] Onasemnogene abeparvovec works by providing a new copy of the gene that makes the human SMN protein.[3]

Onasemnogene abeparvovec was approved for medical use in the United States in 2019, and in Japan and the European Union in 2020.[3][5][4][6]

Onasemnogene abeparvovec has been developed to treat spinal muscular atrophy, a disease linked to a mutation in the SMN1 gene on chromosome 5q[7] and diagnosed predominantly in young children that causes progressive loss of muscle function and frequently death. The medication is administered as an intravenous infusion.

The treatment is approved in the United States and certain other countries for use in children with SMA up to the age of two, including at the presymptomatic stage of the disease.[5]

In the European Union it is indicated for the treatment of patients with SMA who either have a clinical diagnosis of SMA type 1 or have up to three copies of the SMN2 gene.[4]

The medication is used with corticosteroids in an effort to protect the liver.[3]

While marketed as a one-time treatment for SMA, it is unknown how long the onasemnogene abeparvovec-delivered transgene will persist in people. Since motor neurons do not divide, it is expected that the transgene may have long-term stability.[8][failed verification]

Common adverse reactions may include nausea and increased liver enzymes.[3] Serious adverse reactions may include liver problems and low platelets.[3] Transient elevated levels of cardiac troponin‑I were observed in clinical trials; the clinical importance of these findings is not known.[3] However, cardiac toxicity was seen in studies of other animals.[3]

As the medication may reduce the platelet count, platelets may need to be checked before the medication is started, then weekly for the first month and every two weeks for the next two months until the level is back to baseline.[3] Liver function should be monitored for three months after administration.[9]

SMA is a neuromuscular disorder caused by a mutation in the SMN1 gene, which leads to a decrease in SMN protein, a protein necessary for survival of motor neurons. Onasemnogene abeparvovec is a biologic drug consisting of AAV9 virus capsids that contains a SMN1 transgene along with synthetic promoters.[2] Upon administration, the AAV9 viral vector delivers the SMN1 transgene to the affected motor neurons, where it leads to an increase in SMN protein.

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