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Alternate Names: nusinersen sodium, Spinraza
CAS No: 1258984-36-9
PubChem CID: 131801471
Mol Formula: C234H340N61O128P17S17
Mol Weight: 7127.0 g/mol
IUPAC Name: 1-[(2R,3R,4R,5R)-4-[[(2R,3R,4R,5R)-5-(4-amino-5-methyl-2-oxopyrimidin-1-yl)-3-[[(2R,3R,4R,5R)-3-[[(2R,3R,4R,5R)-5-(4-amino-5-methyl-2-oxopyrimidin-1-y -Show More
Commercial / Development Status: Commercial APIs
Source / Manufacturing Origin: Peptide APIs
API Description: Nusinersen (brand name Spinraza) is an antisense oligonucleotide (ASO) therapy designed to treat spinal muscular atrophy (SMA) by increasing production of functional survival motor neuron (SMN) protein. It is administered via intrathecal injection into the cerebrospinal fluid (CSF) to treat pediatric and adult patients, improving motor function by modifying SMN2 gene splicing.
