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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: martin as. 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.
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: martin as. 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.
Noncontiguous operon atlas for the Staphylococcus aureus genome.
Iturbe P, Martín AS, Hamamoto H, Marcet-Houben M, Galbaldón T, Solano C, Lasa I. Iturbe P, et al. Among authors: martin as. Microlife. 2024 Mar 30;5:uqae007. doi: 10.1093/femsml/uqae007. eCollection 2024. Microlife. 2024. PMID: 38651166 Free PMC article.
Draft Guidance for Industry Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, and Related Dystrophinopathies - Developing Potential Treatments for the Entire Spectrum of Disease.
McDonald C, Camino E, Escandon R, Finkel RS, Fischer R, Flanigan K, Furlong P, Juhasz R, Martin AS, Villa C, Sweeney HL. McDonald C, et al. Among authors: martin as. J Neuromuscul Dis. 2024;11(2):499-523. doi: 10.3233/JND-230219. J Neuromuscul Dis. 2024. PMID: 38363616 Free PMC article.
174 results