Nusinersen sodium

• CAT Number: I040121
• Molecular Formula: C234H322N61Na18O128P17S17
• Molecular Weight: 7522.85
• Purity: ≥95%

Nusinersen sodium (Cat No.: I040121) is an antisense oligonucleotide therapy developed for the treatment of spinal muscular atrophy (SMA), a genetic disorder characterized by the loss of motor neurons and progressive muscle weakness. Nusinersen works by promoting the inclusion of exon 7 in the SMN2 gene, leading to the production of functional survival motor neuron (SMN) protein, which is deficient in SMA patients. By increasing SMN protein levels, nusinersen helps to improve motor function and slow disease progression in individuals with SMA, particularly in pediatric and adult patients.

Reference

[1]. Richard S Finkel, et al. Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy. N Engl J Med. 2017 Nov 2;377(18):1723-1732.
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[2]. Laura Torres-Benito, et al. NCALD Antisense Oligonucleotide Therapy in Addition to Nusinersen further Ameliorates Spinal Muscular Atrophy in Mice. Am J Hum Genet. 2019 Jul 3;105(1):221-230.
[Content Brief]