Ataluren (PTC124)

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Chemical Information

Product name：Ataluren (PTC124)	Purity：98% min
CAS NO：775304-57-9	Solubility：DMSO : ≥ 52 mg/mL (182.94 mM)
Molecular Formula：C₁₅H₉FN₂O₃	Package：Package according to customer requirements
Molecular Weight：284.242	Storage：Store at -20℃

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Remarks

Ataluren, also known as PTC124, is an investigational new drug designed to enable the formation of a functioning protein in patients with genetic disorders due to a nonsense mutation. A nonsense mutation is an alteration in the genetic code that prematurely halts the synthesis of an essential protein. Ataluren is currently being investigated for use in patients with nonsense mutation Duchenne/Becker muscular dystrophy (nmDBMD) and cystic fibrosis (nmCF).

Properties:

Density: 1.379

Boiling Point: 503.7ºC at 760 mmHg

Refractive Index: 1.603

CFTR Inhibitors Related Prodcuts:

IOWH-032; PPQ-102; Lumacaftor; Ivacaftor

References

[1]. Pettit RS, et al. CFTR Modulators for the Treatment of Cystic Fibrosis. P T. 2014 Jul;39(7):500-11.

[2]. Welch EM, et al. PTC124 targets genetic disorders caused by nonsense mutations. Nature, 2007, 447(7140), 87-91.

[3]. Miller JN, et al. The novel Cln1(R151X) mouse model of infantile neuronal ceroid lipofuscinosis (INCL) for testing nonsense suppression therapy. Hum Mol Genet. 2015 Jan 1;24(1):185-96.