Risdiplam is a survival of motor neuron-2 (SMN2) splicing modifier.{50413} It enhances SMN2 splicing (EC1.5x = 40 nM) and increases SMN protein levels (EC1.5x = 163 nM) in cellular assays. Risdiplam (3 mg/kg) increases brain and quadricep muscle SMN protein levels in the adult C/C-allele mouse model of mild spinal muscular atrophy (SMA) and the SMND7 neonate mouse model of severe SMA. It also increases the number of vesicular glutamate transporter 1 (vGLUT1) proprioceptive inputs on motor neurons, the number of L3 to L5 spinal motor neurons, the percentage of fully innervated neuromuscular junctions (NMJs), and the size of the extensor digitorum longus muscles in adult SMND7 mice. Formulations containing risdiplam have been used in the treatment of spinal muscular atrophy.
Technical Information
Formal Name
N-(amino-15N-imino-15N-methyl-13C)-2,6-dichloro-benzeneacetamide-15N
CAS Number
1189924-28-4
Molecular Formula
C8[13C]H9Cl2[15N]3O
Formula Weight
250.1
Purity
95%
Formulation
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A solid
Solubility
(Learn about Variance in Solubility)
Methanol: slightly, sonicated
SMILES
ClC1=C(CC([15NH][13C]([15NH2])=[15NH])=O)C(Cl)=CC=C1
InChi Code
InChI=1S/C9H9Cl2N3O/c10-6-2-1-3-7(11)5(6)4-8(15)14-9(12)13/h1-3H,4H2,(H4,12,13,14,15)/i9+1,12+1,13+1,14+1
InChi Key
INJOMKTZOLKMBF-DATIIYMNSA-N
Shipping & Storage Information
Storage -20°C
Shipping
Room Temperature in continental US; may vary elsewhere
Stability
Shelf life: 4 years