Risdiplam  (Synonyms: RG7916; RO7034067)

Risdiplam (RG7916) is an orally administered, centrally and peripherally distributed SMN2 pre-mRNA splicing modifier that increases survival motor neuron (SMN) protein levels^[1].

SMN2

Risdiplam modulates SMN2 pre-mRNA splicing towards the production of full-length SMN2 mRNA and increases SMN protein levels. Risdiplam is a modifier of SMN2 splicing, leading to an increase in SMN2 full length transcript and thus functional SMN protein. Spinal muscular atrophy (SMA) type I remains the most common genetic disease resulting in death in infancy. Characterized by progressive motor and respiratory muscle weakness, this autosomal recessive neuromuscular disorder is caused by low levels of the survival motor neuron protein (SMN) due to inactivating bi-allelic deletions and other disabling mutations in the survival motor neuron 1 (SMN1) gene^[1].

MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.

401.46

C₂₂H₂₃N₇O

1825352-65-5

Solid

White to yellow

O=C(C=C(C(C=C1C)=NN2C1=NC(C)=C2)N=C3C=C4)N3C=C4N(CCN5)CC65CC6

Room temperature in continental US; may vary elsewhere.

* Please refer to the solubility information to select the appropriate solvent. Once prepared, please aliquot and store the solution to prevent product inactivation from repeated freeze-thaw cycles.
Storage method and period of stock solution: -80°C, 6 months; -20°C, 1 month. When stored at -80°C, please use it within 6 months. When stored at -20°C, please use it within 1 month.

• [1]. Poirier A, et al. Risdiplam distributes and increases SMN protein in both the central nervous system and peripheral organs. Pharmacol Res Perspect. 2018 Nov 29;6(6):e00447.  [Content Brief]