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
Davidson D, Stevenson M, Llonch MV, Hsieh A, Brown TM, Slota C, Fehnel S. Development of a novel patient-reported outcome measure symptoms and impacts of familial chylomicronemia syndrome. J Clin Lipidol. 2020 Jul;14(4):P571.