RG7800 hydrochloride (Synonyms: RO6885247 hydrochloride) |
| Catalog No.GC37521 |
RG7800 염산염은 SMN2 스플라이싱 및 SMN 단백질에 대해 EC1.5xs가 23nM 및 87nM인 경구 활성 SMN2 스플라이싱 조절제입니다. RG7800 염산염은 척수성 근위축증을 치료할 가능성이 있습니다.
Products are for research use only. Not for human use. We do not sell to patients.
Sample solution is provided at 25 µL, 10mM.
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RG7800 hydrochloride is an orally active SMN2 splicing modulator, with EC1.5xs of 23 nM and 87 nM for SMN2 splicing and SMN protein; RG7800 hydrochloride has the potential to treat spinal muscular atrophy. EC1.5x: 23 nM (SMN2 splicing), 87 nM (SMN protein)[1]
RG7800 hydrochloride is an orally active SMN2 splicing modulator, with EC1.5xs 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[1].
RG7800 is a favorable agent metabolism and pharmacokinetic (DMPK) profile in the rat and in cynomolgus monkey with good oral bioavailability. RG7800 (1, 3, 10 mg/kg, p.o.) dose-dependently elevates the SMN protein level in the brain and in peripheral tissue of δ7 mice[1].
[1]. Ratni H, et al. Specific Correction of Alternative Survival Motor Neuron 2 Splicing by Small Molecules: Discovery of a Potential Novel Medicine To Treat Spinal Muscular Atrophy. J Med Chem. 2016 Jul 14;59(13):6086-100.
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