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2003 1
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12 results

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Page 1
Hematologic Cancer after Gene Therapy for Cerebral Adrenoleukodystrophy.
Duncan CN, Bledsoe JR, Grzywacz B, Beckman A, Bonner M, Eichler FS, Kühl JS, Harris MH, Slauson S, Colvin RA, Prasad VK, Downey GF, Pierciey FJ, Kinney MA, Foos M, Lodaya A, Floro N, Parsons G, Dietz AC, Gupta AO, Orchard PJ, Thakar HL, Williams DA. Duncan CN, et al. Among authors: downey gf. N Engl J Med. 2024 Oct 10;391(14):1287-1301. doi: 10.1056/NEJMoa2405541. N Engl J Med. 2024. PMID: 39383458 Clinical Trial.
Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy.
Eichler F, Duncan CN, Musolino PL, Lund TC, Gupta AO, De Oliveira S, Thrasher AJ, Aubourg P, Kühl JS, Loes DJ, Amartino H, Smith N, Folloni Fernandes J, Sevin C, Sankar R, Hussain SA, Gissen P, Dalle JH, Platzbecker U, Downey GF, McNeil E, Demopoulos L, Dietz AC, Thakar HL, Orchard PJ, Williams DA. Eichler F, et al. Among authors: downey gf. N Engl J Med. 2024 Oct 10;391(14):1302-1312. doi: 10.1056/NEJMoa2400442. N Engl J Med. 2024. PMID: 39383459 Clinical Trial.
Lentiviral haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase 1/2 trial and expanded access.
Fumagalli F, Calbi V, Natali Sora MG, Sessa M, Baldoli C, Rancoita PMV, Ciotti F, Sarzana M, Fraschini M, Zambon AA, Acquati S, Redaelli D, Attanasio V, Miglietta S, De Mattia F, Barzaghi F, Ferrua F, Migliavacca M, Tucci F, Gallo V, Del Carro U, Canale S, Spiga I, Lorioli L, Recupero S, Fratini ES, Morena F, Silvani P, Calvi MR, Facchini M, Locatelli S, Corti A, Zancan S, Antonioli G, Farinelli G, Gabaldo M, Garcia-Segovia J, Schwab LC, Downey GF, Filippi M, Cicalese MP, Martino S, Di Serio C, Ciceri F, Bernardo ME, Naldini L, Biffi A, Aiuti A. Fumagalli F, et al. Among authors: downey gf. Lancet. 2022 Jan 22;399(10322):372-383. doi: 10.1016/S0140-6736(21)02017-1. Lancet. 2022. PMID: 35065785 Free PMC article. Clinical Trial.
Recommendations for benefit-risk assessment methodologies and visual representations.
Hughes D, Waddingham E, Mt-Isa S, Goginsky A, Chan E, Downey GF, Hallgreen CE, Hockley KS, Juhaeri J, Lieftucht A, Metcalf MA, Noel RA, Phillips LD, Ashby D, Micaleff A; PROTECT Benefit-Risk Group. Hughes D, et al. Among authors: downey gf. Pharmacoepidemiol Drug Saf. 2016 Mar;25(3):251-62. doi: 10.1002/pds.3958. Epub 2016 Jan 22. Pharmacoepidemiol Drug Saf. 2016. PMID: 26800458 Free article.
Adjustment for continuous confounders: an example of how to prevent residual confounding.
Groenwold RH, Klungel OH, Altman DG, van der Graaf Y, Hoes AW, Moons KG; PROTECT WP2 (Pharmacoepidemiological Research on Outcomes of Therapeutics by a European Consortium, Work Programme 2 [Framework for pharmacoepidemiology studies]). Groenwold RH, et al. CMAJ. 2013 Mar 19;185(5):401-6. doi: 10.1503/cmaj.120592. Epub 2013 Feb 11. CMAJ. 2013. PMID: 23401401 Free PMC article. Review. No abstract available.
Phase IIIb safety results from an expanded-access protocol of talimogene laherparepvec for patients with unresected, stage IIIB-IVM1c melanoma.
Chesney J, Awasthi S, Curti B, Hutchins L, Linette G, Triozzi P, Tan MCB, Brown RE, Nemunaitis J, Whitman E, Windham C, Lutzky J, Downey GF, Batty N, Amatruda T. Chesney J, et al. Among authors: downey gf. Melanoma Res. 2018 Feb;28(1):44-51. doi: 10.1097/CMR.0000000000000399. Melanoma Res. 2018. PMID: 29176501 Clinical Trial.
Randomized study of dual versus single ritonavir-enhanced protease inhibitors for protease inhibitor-experienced patients with HIV.
Collier AC, Tierney C, Downey GF, Eshleman SH, Kashuba A, Klingman K, Vergis EN, Pakes GE, Rooney JF, Rinehart A, Mellors JW; AIDS Clinical Trials Group Protocol A5143 Team. Collier AC, et al. Among authors: downey gf. HIV Clin Trials. 2008 Mar-Apr;9(2):91-102. doi: 10.1310/hct0902-91. HIV Clin Trials. 2008. PMID: 18474494 Clinical Trial.
12 results