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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
Camilleri M, Lembo AJ, Lavins BJ, MacDougall JE, Carson RT, Williams VSL, Nelson LM, Shiff SJ, Currie MG, Kurtz CB, Johnston JM. Comparison of adequate relief with symptom, global, and responder endpoints in linaclotide phase 3 trials in IBS-C. United European Gastroenterol J. 2015 Feb 3;3(1):53-62. doi: 10.1177/2050640614555946