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提問: 有沒有人了解美國研究的那種叫PTC124這種藥物的最新進展 問題補充: 尋藥 医师解答: PTC124藥物是由PTC Therapeutics公司生產的一種能夠修正遺傳突變產生的副作用的藥物。它能夠“欺騙”病人的細胞使其產生有功能的產物。有關的最新進展：1: Mechanism of PTC124 activity in cell-based luciferase assays of nonsense codon suppression.Auld DS, Thorne N, Maguire WF, Inglese J.Proc Natl Acad Sci U S A. 2009 Feb 10. [Epub ahead of print]PMID: 19208811 [PubMed - as supplied by publisher]Related Articles Free article in PMC | at journal site2: Drug developers aim to treat cystic fibrosis through disease modification.Thompson CA.Am J Health Syst Pharm. 2008 Dec 15;65:2310-2. No abstract available. PMID: 19052266 [PubMed - indexed for MEDLINE]Related Articles3: Gunvalson decision sends shockwaves through industry.Allison M.Nat Biotechnol. 2008 Nov;26:1201-2. No abstract available. PMID: 18997740 [PubMed - indexed for MEDLINE]Related Articles4: New approaches to treatment of primary immunodeficiencies: fixing mutations with chemicals.Hu H, Gatti RA.Curr Opin Allergy Clin Immunol. 2008 Dec;8:540-6.PMID: 18978469 [PubMed - in process]Related Articles5: Ignoring the nonsense: a phase II trial in cystic fibrosis.Hyde SC, Gill DR.Lancet. 2008 Aug 30;372:691-2. Epub 2008 Aug 20. No abstract available. PMID: 18722009 [PubMed - indexed for MEDLINE]Related Articles6: Effectiveness of PTC124 treatment of cystic fibrosis caused by nonsense mutations: a prospective phase II trial.Kerem E, Hirawat S, Armoni S, Yaakov Y, Shoseyov D, Cohen M, Nissim-Rafinia M, Blau H, Rivlin J, Aviram M, Elfring GL, Northcutt VJ, Miller LL, Kerem B, Wilschanski M.Lancet. 2008 Aug 30;372:719-27. Epub 2008 Aug 20. PMID: 18722008 [PubMed - indexed for MEDLINE]Related Articles7: Pharmacological probing of type 1 autism.Manev H, Manev R.J Autism Dev Disord. 2008 Aug;38:1400-1. Epub 2008 May 30. No abstract available. PMID: 18512134 [PubMed - indexed for MEDLINE]Related Articles8: What's new in cystic fibrosis? From treating symptoms to correction of the basic defect.Proesmans M, Vermeulen F, De Boeck K.Eur J Pediatr. 2008 Aug;167:839-49. Epub 2008 Apr 4. Review.PMID: 18389279 [PubMed - indexed for MEDLINE]Related Articles9: PTC124 is an orally bioavailable compound that promotes suppression of the human CFTR-G542X nonsense allele in a CF mouse model.Du M, Liu X, Welch EM, Hirawat S, Peltz SW, Bedwell DM.Proc Natl Acad Sci U S A. 2008 Feb 12;105:2064-9. Epub 2008 Feb 6.PMID: 18272502 [PubMed - indexed for MEDLINE]Related Articles Free article in PMC | at journal site10: PTC124, nonsense mutations and Duchenne muscular dystrophy.Wilton S.Neuromuscul Disord. 2007 Oct;17:719-20. No abstract available. PMID: 17952895 [PubMed - indexed for MEDLINE]Related Articles11: Studies point way to new therapeutic prospects for muscular dystrophy.Kuehn BM.JAMA. 2007 Sep 26;298:1385-6. No abstract available. PMID: 17895449 [PubMed - indexed for MEDLINE]Related Articles12: Emerging drug treatments for cystic fibrosis.Zeitlin PL.Expert Opin Emerg Drugs. 2007 May;12:329-36. Review.PMID: 17604505 [PubMed - indexed for MEDLINE]Related Articles13: Chemical biology: ignore the nonsense.Schmitz A, Famulok M.Nature. 2007 May 3;447:42-3. No abstract available. PMID: 17450128 [PubMed - indexed for MEDLINE]Related Articles14: PTC124 targets genetic disorders caused by nonsense mutations.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.Nature. 2007 May 3;447:87-91. Epub 2007 Apr 22. PMID: 17450125 [PubMed - indexed for MEDLINE]Related Articles15: Safety, tolerability, and pharmacokinetics of PTC124, a nonaminoglycoside nonsense mutation suppressor, following single- and multiple-dose administration to healthy male and female adult volunteers.Hirawat S, Welch EM, Elfring GL, Northcutt VJ, Paushkin S, Hwang S, Leonard EM, Almstead NG, Ju W, Peltz SW, Miller LL.J Clin Pharmacol. 2007 Apr;47:430-44.PMID: 17389552 [PubMed - indexed for MEDLINE]Related Articles16: Drug evaluation: PTC-124--a potential treatment of cystic fibrosis and Duchenne muscular dystrophy.Hamed SA.IDrugs. 2006 Nov;9:783-9.PMID: 17096300 [PubMed - indexed for MEDLINE]Related Articles17: Readthrough strategies for stop codons in Duchenne muscular dystrophy.Aurino S, Nigro V.Acta Myol. 2006 Jun;25:5-12. Review.PMID: 17039975 [PubMed - indexed for MEDLINE]Related Articles18: Gateways to clinical trials.Bayes M, Rabasseda X, Prous JR.Methods Find Exp Clin Pharmacol. 2005 Jul-Aug;27:411-61.PMID: 16179960 [PubMed - indexed for MEDLINE