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Publications
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
Ribeiro-Oliveira Jr A, Mol SS, Twiss J, Alencar GA, Miranda PA, Naves LA, Azevedo MF, Basilio FM, Boguszewski CL, Nogueira KC, Vilar L, Koltowska-Haggstrom M, McKenna SP. The brazilian version of the Quality of Life Assessment of Growth Hormone Deficiency in Adults (QoL-AGHDA): four-stage translation and validation. Arq Bras Endocrinol Metabol. 2010 Dec;54(9):833-41.