RG7800 hydrochloride (Synonyms: RO6885247 hydrochloride) |
| カタログ番号GC37521 |
RG7800 塩酸塩は、SMN2 スプライシングおよび SMN タンパク質の EC1.5xs が 23 nM および 87 nM である、経口で活性な SMN2 スプライシング モジュレーターです。 RG7800 塩酸塩は、脊髄性筋萎縮症を治療する可能性があります。
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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