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E. M. Welch, E. R. Barton u. a.: PTC124 targets genetic disorders caused by nonsense mutations. In: Nature. Band 447, Nummer 7140, Mai 2007, S. 87–91, doi:10.1038/nature05756, PMID 17450125.
S. Hirawat, E. M. Welch u. a.: Safety, tolerability, and pharmacokinetics of PTC124, a nonaminoglycoside nonsense mutation suppressor, following single- and multiple-dose administration to healthy male and female adult volunteers. In: Journal of clinical pharmacology. Band 47, Nummer 4, April 2007, S. 430–444, doi:10.1177/0091270006297140, PMID 17389552.
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EMEA/COMP/122996/2005 Rev.2 (Memento vom 26. Dezember 2009 im Internet Archive) (PDF; 121 kB) Beurteilung des Committee for Orphan Medicinal Products (COMP) der Europäischen Arzneimittelagentur, 3. Juli 2007.