Nusinersen
Nusinersen (pronounced noo-sin-er-sen) is a medication used in the treatment of Spinal Muscular Atrophy (SMA). It is marketed under the brand name Spinraza by Biogen.
Etymology
The name "Nusinersen" is derived from the drug's chemical structure. The "Nusin" part of the name is an acronym for "Nucleic acid-based Universal Splicing Inhibitor", while "ersen" is a common suffix for antisense oligonucleotides, a class of drugs to which Nusinersen belongs.
Pharmacology
Nusinersen is an antisense oligonucleotide (ASO) that modifies the splicing of SMN2, a gene that is critical in the pathogenesis of SMA. By altering the splicing of SMN2, Nusinersen increases the production of full-length Survival Motor Neuron (SMN) protein, which is deficient in individuals with SMA.
Usage
Nusinersen is used for the treatment of SMA in pediatric and adult patients. It is administered via intrathecal injection, which delivers the medication directly into the spinal fluid.
Side Effects
Common side effects of Nusinersen include headache, back pain, and vomiting. More serious side effects can include kidney damage and low platelet counts.
Related Terms
External links
- Medical encyclopedia article on Nusinersen
- Wikipedia's article - Nusinersen
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