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Publications
Peay HL, Fischer R, Mange B, Paquin RS, Smith EC, Sadosky A, Russo L, Ricotti V, Rensch C, Morris C, Martin AS, Ganot A, Beaverson K, Mansfield C. Patients' and caregivers' maximum acceptable risk of death for non-curative gene therapy to treat Duchenne muscular dystrophy. Mol Genet Genomic Med. 2021 May;9(5):e1664. doi: 10.1002/mgg3.1664
Pavel M, Horsch D, Anthony L, Ervin C, Kulke MH, Bergsland E, Caplin M, Oberg K, Warner R, Kunz P, Metz DC, Pasieka J, Pavlakis N, DiBenedetti D, Lapuerta P. Patient interviews in TELESTAR, a phase 3 study of telotristat etiprate, report meaningful improvement in carcinoid syndrome. Poster presented at the 2016 European Neuroendocrine Tumor Society (ENETS) Annual Meeting; March 2016. Barcelona, Spain. [abstract] Neuroendocrinology. 2016; 103:89.