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

Eitan Kerem*, Samit Hirawat, Shoshana Armoni, Yasmin Yaakov, David Shoseyov, Michael Cohen, Malka Nissim-Rafinia, Hannah Blau, Joseph Rivlin, Micha Aviram, Gary L. Elfring, Valerie J. Northcutt, Langdon L. Miller, Batsheva Kerem, Michael Wilschanski

*Corresponding author for this work

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Medicine and Dentistry

Biochemistry, Genetics and Molecular Biology

Neuroscience

Pharmacology, Toxicology and Pharmaceutical Science