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
Pelletier SJ, Fu S, Thyagarajan (Hoffman) V, Romero-Marrero C, Batheja MJ, Punch JD, Magee JC, Lok AS, Fontana RJ, Marrero JA. An intention-to-treat analysis of liver transplantation for hepatocellular carcinoma using organ procurement transplant network data. Liver Transpl Surg. 2009 Aug;15(8):859-68. doi: 10.1002/lt.21778
McKenna SP, Doward LC. How should we measure quality of life in ankylosing spondylitis? Rheumatol Pract. 2008;6(2):12-4.