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Page 1
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: dietz ac. N Engl J Med. 2024 Oct 10;391(14):1302-1312. doi: 10.1056/NEJMoa2400442. N Engl J Med. 2024. PMID: 39383459 Clinical Trial.
International workshop: what is needed to ensure outcome measures for Rett syndrome are fit-for-purpose for clinical trials? June 7, 2023, Nashville, USA.
Downs J, Pichard DC, Kaufmann WE, Horrigan JP, Raspa M, Townend G, Marsh ED, Leonard H, Motil K, Dietz AC, Garg N, Ananth A, Byiers B, Peters S, Beatty C, Symons F, Jacobs A, Youakim J, Suter B, Santosh P, Neul JL, Benke TA. Downs J, et al. Among authors: dietz ac. Trials. 2024 Dec 21;25(1):845. doi: 10.1186/s13063-024-08678-6. Trials. 2024. PMID: 39709426 Free PMC article.
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: dietz ac. N Engl J Med. 2024 Oct 10;391(14):1287-1301. doi: 10.1056/NEJMoa2405541. N Engl J Med. 2024. PMID: 39383458 Clinical Trial.
Recommendations for surveillance of pulmonary dysfunction among childhood, adolescent, and young adult cancer survivors: a report from the International Late Effects of Childhood Cancer Guideline Harmonization Group.
Otth M, Kasteler R, Mulder RL, Agrusa J, Armenian SH, Barnea D, Bergeron A, Bhatt NS, Bourke SJ, Constine LS, Goutaki M, Green DM, Hennewig U, Houdouin V, Hudson MM, Kremer L, Latzin P, Ng A, Oeffinger KC, Schindera C, Skinner R, Sommer G, Srinivasan S, Stokes DC, Versluys B, Waespe N, Weiner DJ, Dietz AC, Kuehni CE. Otth M, et al. Among authors: dietz ac. EClinicalMedicine. 2024 Feb 20;69:102487. doi: 10.1016/j.eclinm.2024.102487. eCollection 2024 Mar. EClinicalMedicine. 2024. PMID: 38420219 Free PMC article. Review.
Variables affecting outcomes after allogeneic hematopoietic stem cell transplant for cerebral adrenoleukodystrophy.
Chiesa R, Boelens JJ, Duncan CN, Kühl JS, Sevin C, Kapoor N, Prasad VK, Lindemans CA, Jones SA, Amartino HM, Algeri M, Bunin N, Diaz-de-Heredia C, Loes DJ, Shamir E, Timm A, McNeil E, Dietz AC, Orchard PJ. Chiesa R, et al. Among authors: dietz ac. Blood Adv. 2022 Mar 8;6(5):1512-1524. doi: 10.1182/bloodadvances.2021005294. Blood Adv. 2022. PMID: 34781360 Free PMC article.
Lung Cancer as a Subsequent Malignant Neoplasm in Survivors of Childhood Cancer.
Ghosh T, Chen Y, Dietz AC, Armstrong GT, Howell RM, Smith SA, Mulrooney DA, Turcotte LM, Yuan Y, Yasui Y, Neglia JP. Ghosh T, et al. Among authors: dietz ac. Cancer Epidemiol Biomarkers Prev. 2021 Dec;30(12):2235-2243. doi: 10.1158/1055-9965.EPI-21-0250. Epub 2021 Sep 14. Cancer Epidemiol Biomarkers Prev. 2021. PMID: 34526300 Free PMC article.
Pooled safety analysis of tisagenlecleucel in children and young adults with B cell acute lymphoblastic leukemia.
Levine JE, Grupp SA, Pulsipher MA, Dietz AC, Rives S, Myers GD, August KJ, Verneris MR, Buechner J, Laetsch TW, Bittencourt H, Baruchel A, Boyer MW, De Moerloose B, Qayed M, Davies SM, Phillips CL, Driscoll TA, Bader P, Schlis K, Wood PA, Mody R, Yi L, Leung M, Eldjerou LK, June CH, Maude SL. Levine JE, et al. Among authors: dietz ac. J Immunother Cancer. 2021 Aug;9(8):e002287. doi: 10.1136/jitc-2020-002287. J Immunother Cancer. 2021. PMID: 34353848 Free PMC article.
48 results