About: PTC124

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An Entity of Type : http://linked.opendata.cz/ontology/drugbank/Drug, within Data Space : linked.opendata.cz associated with source document(s)

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rdf:type	http://linked.opendata.cz/ontology/drugbank/Drug
http://linked.open...gbank/description	PTC124 is a novel, orally administered drug that targets nonsense mutations and is being investigated initially as a treatment for Duchenne muscular dystrophy (DMD) and cystic fibrosis (CF), with the potential to treat a number of other genetic disorders caused by nonsense mutations. (en)
http://linked.open...generalReferences	# Welch EM, Barton ER, Zhuo J, Tomizawa Y, Friesen WJ, Trifillis P, Paushkin S, Patel M, Trotta CR, Hwang S, Wilde RG, Karp G, Takasugi J, Chen G, Jones S, Ren H, Moon YC, Corson D, Turpoff AA, Campbell JA, Conn MM, Khan A, Almstead NG, Hedrick J, Mollin A, Risher N, Weetall M, Yeh S, Branstrom AA, Colacino JM, Babiak J, Ju WD, Hirawat S, Northcutt VJ, Miller LL, Spatrick P, He F, Kawana M, Feng H, Jacobson A, Peltz SW, Sweeney HL: PTC124 targets genetic disorders caused by nonsense mutations. Nature. 2007 May 3;447(7140):87-91. Epub 2007 Apr 22. "Pubmed":http://www.ncbi.nlm.nih.gov/pubmed/17450125 # Hirawat S, Welch EM, Elfring GL, Northcutt VJ, Paushkin S, Hwang S, Leonard EM, Almstead NG, Ju W, Peltz SW, Miller LL: Safety, tolerability, and pharmacokinetics of PTC124, a nonaminoglycoside nonsense mutation suppressor, following single- and multiple-dose administration to healthy male and female adult volunteers. J Clin Pharmacol. 2007 Apr;47(4):430-44. "Pubmed":http://www.ncbi.nlm.nih.gov/pubmed/17389552 # Hamed SA: Drug evaluation: PTC-124--a potential treatment of cystic fibrosis and Duchenne muscular dystrophy. IDrugs. 2006 Nov;9(11):783-9. "Pubmed":http://www.ncbi.nlm.nih.gov/pubmed/17096300 (en)
http://linked.open...gy/drugbank/group	investigational (en)
http://linked.open...drugbank/halfLife	3-6 hours (en)
http://linked.open...ugbank/indication	Investigated for use/treatment in cystic fibrosis and muscular dystrophy. (en)
sameAs	PTC124
Title	PTC124 (en)
adms:identifier	http://linked.opendata.cz/resource/drugbank/drug/DB05016/identifier/chemspider/9394889 http://linked.opendata.cz/resource/drugbank/drug/DB05016/identifier/drugbank/DB05016 http://linked.opendata.cz/resource/drugbank/drug/DB05016/identifier/pubchem-compound/11219835 http://linked.opendata.cz/resource/drugbank/drug/DB05016/identifier/pubchem-substance/29203630
http://linked.open...mechanismOfAction	PTC124 allowed the cellular machinery to bypass the nonsense mutation, continue the translation process, and thereby restore the production of a full-length, functional protein. The research on the effects of PTC124 on the translation and stability of nonsense-containing mRNA in vitor show that PTC124 promoted readthrough at each of the nonsense codons, showing maximal activity with UGA, while having no effect on mRNA levels. Unlike the stable cell line assays, PTC124 did not discriminate significantly between the UAG and UAA mRNAs. PTC124 was a more potent nonsense-suppressing agent than gentamicin, and exhibited 4- to 15-fold stimulation of in vitro readthrough relative to the controls at levels similar to those in the stable cell reporter assays. These results indicate that PTC124 modulates termination efficiency at premature nonsense codons. (en)
http://linked.open.../drugbank/synonym	EC-000.2051 (en)
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