Effectiveness of PTC124 treatment of cystic fibrosis caused by nonsense mutations: a prospective phase II trial

Kerem, Eitan, Dr ; Hirawat, Samit, MD ; Armoni, Shoshana, BSN ; Yaakov, Yasmin, MSc ; Shoseyov, David, MD ; Cohen, Michael, MD ; Nissim-Rafinia, Malka, PhD ; Blau, Hannah, MD ; Rivlin, Joseph, MD ; Aviram, Micha, MD ; Elfring, Gary L, MS ; Northcutt, Valerie J, MS ; Miller, Langdon L, MD ; Kerem, Batsheva, PhD ; Wilschanski, Michael, MBBS

The Lancet (British edition), 2008-08, Vol.372 (9640), p.719-727 [Periódico revisado por pares]

England: Elsevier Ltd

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