Ligand id: 9416

Name: nusinersen

Structure and Physico-chemical Properties

2D Structure
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Calculated Physico-chemical Properties
Hydrogen bond acceptors 161
Hydrogen bond donors 23
Rotatable bonds 176
Topological polar surface area 3144.17
Molecular weight 7122.28
XLogP -19.23
No. Lipinski's rules broken 3

Molecular properties generated using the CDK

No information available.
Summary of Clinical Use
Nusinersen was approved in December 2016 for the treatment of spinal muscular atrophy (SMA) in pediatric [4-5] and adult patients. Phase 1 trial results were reported in [2] and Phase 2 results in [3]. Nusinersen is administered directly to the central nervous system via intrathecal injection.
Mechanism Of Action and Pharmacodynamic Effects
Nusinersen improves production of the SMN2 protein [7], whose loss causes the neuromuscular disease SMA. Nusinersen restores proper splicing of exon 7 which is abnormally spliced-out in SMA patients. Loss of exon 7 results in a truncated SMN2 protein (SMNdelta7 protein) which is unstable and inactive, leading to reduced levels of functional SMN protein. Nusinersen-induced restoration of SMN2 protein levels provides therapeutic benefit. The molecular mechanism underlying the ability of ASOs to modulate alternative splicing was originally described in [8].
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