Description
PTC124 allows read-through of premature stop codons in mRNA, enabling proper protein translation. This compound is used to treat Duchenne muscular dystrophy (DMD) and cystic fibrosis as well as other nonsense mutation disorders.
PTC124 allows read-through of premature stop codons in mRNA, enabling proper protein translation. This compound is used to treat Duchenne muscular dystrophy (DMD) and cystic fibrosis as well as other nonsense mutation disorders.
| Cas No. | 775304-57-9 |
|---|---|
| Purity | ≥98% |
| Formula | C15H9FN2O3 |
| Formula Wt. | 284.25 |
| IUPAC Name | 3-[5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl]benzoic acid |
| Synonym | PTC-124, Ataluren, Translarna™ |
| Solubility | DMSO 57 mg/mL (200mM) Water Insoluble Ethanol Insoluble |
| Store Temp | -20°C |
|---|---|
| Ship Temp | Ambient |
| MSDS | |
|---|---|
| Info Sheet |
Bushby K, Finkel R, Wong B, et al. Ataluren treatment of patients with nonsense mutation dystrophinopathy. Muscle Nerve. 2014 Oct;50(4):477-87. PMID: 25042182.
Ryan NJ. Ataluren: first global approval. Drugs. 2014 Sep;74(14):1709-14. PMID: 25193627.
Nagel-Wolfrum K, Möller F, Penner I, et al. Translational read-through as an alternative approach for ocular gene therapy of retinal dystrophies caused by in-frame nonsense mutations. Vis Neurosci. 2014 Sep;31(4-5):309-16. PMID: 24912600.
Peptide found in insects.
Polyene macrolide, induces pore formation in fu...
Nucleotide analog; RT inhibitor.
Folic acid derivative
Endogenous neuropeptide hormone, lowers extrace...
Organosulfur, antioxidant.
Endogenous peptide, produced by activation of P...
Nonpeptide angiotensin II receptor antagonist
Sulfonylurea; ATP-sensitive K+ channel blocker....
Glucosinolate found in cruciferous vegetables.<...
NSAID; COX-2 inhibitor.
Isoflavone, phytoestrogen found in soy
Indirubin derivative.
May be useful against COVID-19
Piperazine; 5-HT1 agonist, 5-HT2/3 and α2-adre...
Steroid lactone produced by Withania somnifera;...
Proteinase subtrate.
Thiol prodrug, antioxidant.
Benzophenanthridine alkaloid found in Garcinia....
STING inhibitor