Chemical Name : MLN8237
CAS : 1028486-01-2
Synonyms: 4-[[9-Chloro-7-(2-fluoro-6-methoxyphenyl)-5H-pyrimido[5,4-d]benzazepin-2-yl]amino]-2-methoxybenzoic acid
Solubility:Soluble in DMSO (5mg/ml)
Storage:-20C 2 years
Treat Cystic Fibrosis and Duchenne and Becker Muscular Dystrophy (nmDMD/BMD). It shows promising result in Phase 2. Orally bioavailable 284 D-1,2,4-oxadiazole. Promotes readthrough of premature translation termination codons. Targets genetic disorders caused by nonsense mutations. Shows no concomitant effects on normal termination or mRNA decay.
1��PTC124 targets genetic disorders caused by nonsense mutations: E.M. Welch, et al.; Nature 447, 87 (2007) 2��PTC124 is an orally bioavailable compound that promotes suppression of the human CFTR-G542X nonsense allele in a CF mouse model: M. Du, et al.; PNAS 105, 2064 (2008) 3��Effectiveness of PTC124 treatment of cystic fibrosis caused by nonsense mutations: a prospective phase II trial: E. Kerem, et al.; Lancet 372, 719 (2008)