Onasemnogene abeparvovec
Onasemnogene Abeparvovec
Onasemnogene abeparvovec (pronunciation: oh-na-sem-no-jeen a-be-par-vo-vec), also known as Zolgensma, is a gene therapy medication used for the treatment of Spinal Muscular Atrophy (SMA).
Etymology
The name "Onasemnogene abeparvovec" is derived from the Greek words "on" meaning "in", "sema" meaning "sign", "gene" meaning "origin", "abe" meaning "life", "parvo" meaning "small", and "vec" meaning "vehicle". This refers to the medication's function as a small vehicle carrying life-originating signs into the body.
Usage
Onasemnogene abeparvovec is used to treat spinal muscular atrophy, a genetic disorder characterized by the loss of motor neurons and progressive muscle wasting. It is specifically indicated for pediatric patients less than 2 years of age with SMA.
Mechanism of Action
Onasemnogene abeparvovec works by delivering a fully functional copy of the human SMN gene into the target motor neuron cells. This is achieved through the use of a viral vector, specifically an Adeno-associated virus (AAV9) vector.
Related Terms
External links
- Medical encyclopedia article on Onasemnogene abeparvovec
- Wikipedia's article - Onasemnogene abeparvovec
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