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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
Noel E, Dussol B, Lacombe D, Bedreddine N, Fouilhoux A, Ronco P, Genevaz D, Bekri S, Hagege A, Dupuis-Simeon F, Derrien V, Germain DP, Lidove O. Fabry patients' needs and expectations regarding their treatment in France: development of a Patients' Need Questionnaire (PNQ Fabry). Poster presented at the 2019 WORLDSymposium; February 2019. Orlando, FL. [abstract] Mol Genet Metab. 2019 Feb; 126(2):S110. doi: 10.1016/j.ymgme.2018.12.278