Synonyms: PTC124
ataluren is an approved drug (EMA (2014))
Compound class:
Synthetic organic
Comment: Ataluren is a compound in an emerging class of chemicals which induce ribosomal readthrough. This developing pharmaceutical intervention has been termed 'readthrough strategy'. Clinically, this type of compound has potential benefit in the treatment of genetic disorders associated with loss of functional protein due to nonsense or PTC mutations, such as certain types of muscular dystrophy, cystic fibrosis and many cancers [1,4].
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References |
1. Ainsworth C. (2005)
Nonsense mutations: running the red light. Nature, 438 (7069): 726-8. [PMID:16340982] |
2. Hoffman EP, Connor EM. (2013)
Orphan drug development in muscular dystrophy: update on two large clinical trials of dystrophin rescue therapies. Discovery medicine, 16 (89): 33-9. [PMID:24229740] |
3. McElroy SP, Nomura T, Torrie LS, Warbrick E, Gartner U, Wood G, McLean WH. (2013)
A lack of premature termination codon read-through efficacy of PTC124 (Ataluren) in a diverse array of reporter assays. PLoS biology, 11 (6): e1001593. [PMID:23824517] |
4. Welch EM, Barton ER, Zhuo J, Tomizawa Y, Friesen WJ, Trifillis P, Paushkin S, Patel M, Trotta CR, Hwang S et al. (2007)
PTC124 targets genetic disorders caused by nonsense mutations. Nature, 447 (7140): 87-91. [PMID:17450125] |