- PTC124
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ImageFile=PTC124.svg
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IUPACName=3- [5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl] benzoic acid
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Section1= Chembox Identifiers
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PubChem=11219835
SMILES=C1=CC=C(C(=C1)C2=NC(=NO2)C3=CC(=CC=C3)C(=O)O)F
Section2= Chembox Properties
Formula=C15H9FN2O3
MolarMass=284.241963
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Section3= Chembox Hazards
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Autoignition=PTC124 is a novel small-molecular agent that makes
ribosome s less sensitive tostop codon s. However, it is not equally effective with every stop codon, working best on the sequence 'UGA' cite journal |author=Welch EM, Barton ER, Zhuo J, "et al" |title=PTC124 targets genetic disorders caused by nonsense mutations |journal=Nature |volume=447 |issue=7140 |pages=87–91 |year=2007 |pmid=17450125 |doi=10.1038/nature05756] .It has been investigated for use against conditions caused by
nonsense mutations cite journal |author=Hirawat S, Welch EM, Elfring GL, "et al" |title=Safety, tolerability, and pharmacokinetics of PTC124, a nonaminoglycoside nonsense mutation suppressor, following single- and multiple-dose administration to healthy male and female adult volunteers |journal=Journal of clinical pharmacology |volume=47 |issue=4 |pages=430–44 |year=2007 |pmid=17389552 |doi=10.1177/0091270006297140] , such asDuchenne muscular dystrophy andcystic fibrosis .PTC124 is scheduled to enter the final phase of clinical trials in 2007. [cite web |url=http://www.timesonline.co.uk/tol/news/uk/science/article1690544.ece |title=Daily pill to beat genetic diseases - Times Online |accessdate=2007-10-23 |format= |work=]
It has been developed by
PTC Therapeutics .cite journal |author=Hamed SA |title=Drug evaluation: PTC-124--a potential treatment of cystic fibrosis and Duchenne muscular dystrophy |journal=IDrugs : the investigational drugs journal |volume=9 |issue=11 |pages=783–9 |year=2006 |pmid=17096300 |doi=]References
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