What is Onasemnogene abeparvovec Zolgensma
Onasemnogene abeparvovec-zolgensma is a gene therapy treatment for spinal muscular atrophy (SMA). It is a single administration treatment that replaces the defective SMN1 gene with a healthy copy, restoring production of the SMN protein. This protein is essential for normal muscle function, and people with SMA lack it due to a genetic defect. Onasemnogene abeparvovec-zolgensma is the first gene therapy treatment for SMA approved by the US Food and Drug Administration (FDA).
Benefits of Onasemnogene abeparvovec Zolgensma
and Its Uses.
Onasemnogene abeparvovec (trade name Zolgensma) is a gene therapy medication used to treat a rare form of muscular dystrophy called Spinal Muscular Atrophy (SMA) in infants and children under two years old. SMA is a genetic disorder that results in the loss of nerve cells in the spinal cord, causing muscle weakness and atrophy. Zolgensma is a single-dose gene therapy that delivers a working copy of the SMN1 gene to cells that produce the defective SMN protein, restoring muscle function.
Zolgensma was approved by the U.S. Food and Drug Administration (FDA) in May 2019 for the treatment of SMA in infants and children under two years old. It is the first and only gene therapy approved for use in SMA.
Zolgensma is a life-saving treatment for infants and children with SMA, a rare and
Side Effects and Dosage of Onasemnogene abeparvovec Zolgensma
Onasemnogene abeparvovec (Zolgensma) is a gene therapy drug used to treat spinal muscular atrophy (SMA). It is a one-time infusion that replaces the defective gene that causes SMA.
Onasemnogene abeparvovec is a prescription drug and should be taken as directed by your doctor.
There are no known side effects of Onasemnogene abeparvovec. However, as with any drug, there is always a risk of side effects. If you experience any side effects after taking Onasemnogene abeparvovec, contact your doctor immediately.
Onasemnogene abeparvovec is not recommended for pregnant or breastfeeding women. It is also not recommended for children under 2 years of age.
Onasemnogene abeparvovec is a very expensive drug. The cost of the drug may vary depending on your insurance