PTC124

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
CASNo=
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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Density=
MeltingPt=
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Section3= Chembox Hazards
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PTC124 is a novel small-molecular agent that makes ribosomes less sensitive to stop codons. 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 mutationscite 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 as Duchenne muscular dystrophy and cystic 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=]

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Look at other dictionaries:

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  • Duchenne muscular dystrophy — Classification and external resources Histopathology of gastrocnemius muscle from patient who died of pseudohypertrophic muscular dystrophy, Duchenne type. Cross section of muscle shows extensive replacement of muscle fibers by adipose cells …   Wikipedia

  • PTC Therapeutics — is a pharmaceutical company. It is headquartered in South Plainfield, New Jersey.They are the company responsible for the development of PTC124.cite journal |author=Hamed SA |title=Drug evaluation: PTC 124 a potential treatment of cystic fibrosis …   Wikipedia

  • PTC-124 — Strukturformel Allgemeines Name PTC 124 Andere Namen 3 [5 (2 Fluor phenyl) [1,2,4]oxadiazol 3 yl] benzoesäure (IUPAC) …   Deutsch Wikipedia

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