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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
Garland J, Stephen J, Class B, Gruber A, Ciccone C, Poliak A, Hayes CP, Singhal V, Slota C, Perreault J, Gavrilova R, Shrader JA, Chittiboina P, Joe G, Heiss J, Gahl WA, Huizing M, Carrillo N, Malicdan MCV. Identification of an Alu element-mediated deletion in the promoter region of GNE in siblings with GNE myopathy. Mol Genet Genomic Med. 2017 Jun 14;5(4):410-7. doi: 10.1002/mgg3.300
Anthony L, Horsch D, Ervin C, Kulke MH, Pavel M, Bergsland E, Caplin M, Oberg K, Warner R, Kunz P, Metz DC, Pasieka J, Pavlakis N, DiBenedetti D, Haydysch E, Yang QM, Jackson S, Arnold K, Law L, Lapuerta P. Assessing treatment benefit of telotristat etiprate in patients with carcinoid syndrome: patient exit interviews. Poster presented at the 2016 UKI NETS 14th National Conference; December 2016. London, UK. [abstract] Pancreas. 2016 Dec; 45(3):470. Previously presented at the 8th Symposium of the North American Neuroendocrine Tumor Society. doi: 10.1530/endoabs.46.P11