RG7800 hydrochloride is an orally active SMN2 splicing modulator, with EC 1.5x s of 23 nM and 87 nM for SMN2 splicing and SMN protein ; RG7800 hydrochloride has the potential to treat spinal muscular atrophy.Appearance:SolidIC50& Target:EC1.5x: 23 nM ( SMN2 splicing), 87 nM (SMN protein)In Vitro:RG7800 hydrochloride is an orally active SMN2 splicing modulator, with EC 1.5x s of 23 nM and 87 nM for SMN2 splicing and SMN protein; and is the first small molecule SMN2 splicing modifier to enter human clinical trials. MCE has not independently coIn Vivo:RG7800 is a favorable agent metabolism and pharmacokinetic (DMPK) profile in the rat and in cynomolgus monkey with good oral bioavailability. RG7800 (1, 3, 10mg/kg, p.o.) dose-dependently elevates the SMN protein level in the brain and in peripheral tissBiological Activity:RG7800 hydrochloride is an orally active SMN2 splicing modulator, with EC 1.5x s of 23 nM and 87 nM for SMN2 splicing and SMN protein ; RG7800 hydrochloride has the potential to treat spinal muscular atrophy. Specifications and Purity: 99%. Molecular Formula: C24H28N6O.xHCl.
- UPC:
- 12352208
- Condition:
- New
- Availability:
- 8-12 weeks
- Weight:
- 1.09 Ounces
- HazmatClass:
- No
- WeightUOM:
- LB
- MPN:
- R659305-5mg
- Product Size:
- 5mg
