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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: mange b. 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: mange b. 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.
Patient-Centered Identification of Meaningful Regulatory Endpoints for Medical Devices to Treat Parkinson's Disease.
Benz HL, Caldwell B, Ruiz JP, Saha A, Ho M, Christopher S, Bardot D, Sheehan M, Donnelly A, McLaughlin L, Mange B, Hauber AB, Gwinn K, Heetderks WJ, Sheldon M. Benz HL, et al. Among authors: mange b. MDM Policy Pract. 2021 Jul 2;6(1):23814683211021380. doi: 10.1177/23814683211021380. eCollection 2021 Jan-Jun. MDM Policy Pract. 2021. PMID: 34277950 Free PMC article.
15 results