European Journal of Biomedical and Pharmaceutical Sciences

ZOLGENSMA (ONASEMNOGENE-ABEPARVOVEC) DRUG USED IN SPINAL MUSCULAR ATROPHY(SMA)

Vignesh S.*, Nandhitha P., Jothimanivannan C., Vinothini A. and Aravinth M.

ABSTRACT

ZOLGENSMA (ONASEMNOGENE ABEPARVOVEC) is a gene therapy used to treat spinal muscular atropy (SMA), a rare genetic disease caused by a problem with the SMN1 gene. This gene helps make a protein needed for muscle strength, and when it doesn’t work, muscles strength, and when it doesn’t work, muscles get weaker over time. Zolgensma works by using a virus (AAV9) to deliver a healthy copy of the SMN1 gene to the muscles, allowing them to produce the needed protein and stop the disease from getting worse. It ‘ s given as a single intravenous infusion and has shown great results, especially in babies with the most severe form of SMA (TYPE1).early treatment can help babies reach important motor milestones, like sitting, standing, and breathing without help. common side effects may include liver damage or low platelet counts. approved by the U.S FDA in 2019, zolgensma is a promising treatment that could significantly improve life for those with SMA, even though it is very expensive.

Keywords: spinal muscular atrophy, zolgensma (onasemnogene abeparvovec), SM1GENE, adeno virus (AAV9).