Skip to main content
Publications
Keens T, Hoffman V, Topuria I, Elder K, Cerf S, Mulder K, Roberts J, Lysinger J, Del Carmen Reyes M, Berdella M, Cairns AM, Jain M, Ganapathy V, Lou Y, Morcos B, Wu C, Sass L, VX19-CFD-003 Study Group. Real-world effectiveness of elexacaftor/tezacaftor/ivacaftor on the burden of illness in adolescents and adults with cystic fibrosis. Heliyon. 2024 Mar 26;10(7):e28508. doi: 10.1016/j.heliyon.2024.e28508
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