Chronic ataluren (PTC124) treatment of nonsense mutation cystic fibrosis
Wilschanski M, Miller LL, Shoseyov D et al.
Hadassah University Hospital, Jerusalem, Israel.
Eur Respir J 2011;38:59–69.
Editor’s note: Ataluren (PTC124) is an orally bioavailable drug that induces selective ribosomal read-through of mRNA containing a premature stop codon, thereby allowing the translation of full-length protein. In the present study, Wilschanski et al. investigate the effects of long-term ataluren treatment in a group of CF patients with stop mutations.
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