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Priorities when deciding on participation in early-phase gene therapy trials for Duchenne muscular dystrophy: a best-worst scaling experiment in caregivers and adult patients.
Paquin RS, Fischer R, Mansfield C, Mange B, Beaverson K, Ganot A, Martin AS, Morris C, Rensch C, Ricotti V, Russo LJ, Sadosky A, Smith EC, Peay HL. Paquin RS, et al. Among authors: ganot a. Orphanet J Rare Dis. 2019 May 9;14(1):102. doi: 10.1186/s13023-019-1069-6. Orphanet J Rare Dis. 2019. PMID: 31072340 Free PMC article.
Patients' and caregivers' maximum acceptable risk of death for non-curative gene therapy to treat Duchenne muscular dystrophy.
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. Peay HL, et al. Among authors: ganot a. Mol Genet Genomic Med. 2021 May;9(5):e1664. doi: 10.1002/mgg3.1664. Epub 2021 Mar 23. Mol Genet Genomic Med. 2021. PMID: 33755338 Free PMC article.
Clinician Perspectives of Gene Therapy as a Treatment Option for Duchenne Muscular Dystrophy.
Cope H, Fischer R, Heslop E, McNiff M, Johnson A, Camino E, Denger B, Armstrong N, Thakrar S, Bateman-House A, Beaverson KL, Woollacott IOC, Phillips D, Fernandez V, Ganot A, Donisa-Dreghici R, Mansfield C, Peay H. Cope H, et al. Among authors: ganot a. J Neuromuscul Dis. 2024;11(5):1085-1093. doi: 10.3233/JND-240033. J Neuromuscul Dis. 2024. PMID: 39093077 Free PMC article.
Clinical potential of microdystrophin as a surrogate endpoint.
Boehler JF, Brown KJ, Beatka M, Gonzalez JP, Donisa Dreghici R, Soustek-Kramer M, McGonigle S, Ganot A, Palmer T, Lowie C, Chamberlain JS, Lawlor MW, Morris CA. Boehler JF, et al. Among authors: ganot a. Neuromuscul Disord. 2023 Jan;33(1):40-49. doi: 10.1016/j.nmd.2022.12.007. Epub 2022 Dec 14. Neuromuscul Disord. 2023. PMID: 36575103 Free article. Review.