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
Frye S, Crosby M, Edwards TP, Juliano R. US academic drug discovery. Nat Rev Drug Discov. 2011 Jun 1;10(6):409-10. doi: 10.1038/nrd3462