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Molecular properties generated using the CDK
|Compound class||Synthetic organic|
|Approved drug?||Yes (source: FDA (2016))|
|International Nonproprietary Names|
|CAS Registry No.||1258984-36-9 (source: WHO INN record)|
|Search Google for chemical match using the InChIKey||WWFDJIVIDXJAQR-FFWSQMGZSA-N|
|Search Google for chemicals with the same backbone||WWFDJIVIDXJAQR|
|Search PubMed clinical trials||nusinersen|
|Search PubMed titles||nusinersen|
|Search PubMed titles/abstracts||nusinersen|
|Nusinersen (ISIS 396443) is an antisense oligo(ribo)nucleotide (ASO) which induces survival of motor neuron (SMN) expression .
Nusinersen is designated as an orphan drug by the US FDA and EMA and is the first drug approved for the treatment of spinal muscular atrophy (SMA).
The sequence contains 2'-O-(2-methoxyethyl) (2'-MOE)-oligoribonucleotides to reduce nuclease degradation and enhance binding affinity towards the complementary RNA. The full sequence is [2'-O-(2-methoxyethyl)](3'-5')(P-thio)(mU-mC-A-mC-mU-mU-mU-mC-A-mU-A-A-mU-G-mC-mU-G-G) as detailed in the INN record for the agent. A full structural rendering using SMILES from Chemspider is available here (CSID:34983394). The sequence for nusinersen is claimed in patent WO2010148249A1 as SEQ ID NO: 1 .