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
Thijssen JJJ, Bregantini D. Costly sequential experimentation and project valuation with an application to health technology assessment. J Econ Dyn Control. 2017 Apr 1;77:202-29. doi: 10.1016/j.jedc.2017.01.016
Brown TM, Horblyuk RV, Grotzinger KM, Matzdorff AC, Pashos CL. Patient-reported treatment burden of chronic immune thrombocytopenia therapies. BMC Blood Disord. 2012 Mar 1;12(1):2.