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2019 3
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15 results

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Page 1
Elexacaftor-Tezacaftor-Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele.
Middleton PG, Mall MA, Dřevínek P, Lands LC, McKone EF, Polineni D, Ramsey BW, Taylor-Cousar JL, Tullis E, Vermeulen F, Marigowda G, McKee CM, Moskowitz SM, Nair N, Savage J, Simard C, Tian S, Waltz D, Xuan F, Rowe SM, Jain R; VX17-445-102 Study Group. Middleton PG, et al. N Engl J Med. 2019 Nov 7;381(19):1809-1819. doi: 10.1056/NEJMoa1908639. Epub 2019 Oct 31. N Engl J Med. 2019. PMID: 31697873 Free PMC article. Clinical Trial.
Tumor inflammation-associated neurotoxicity.
Mahdi J, Dietrich J, Straathof K, Roddie C, Scott BJ, Davidson TB, Prolo LM, Batchelor TT, Campen CJ, Davis KL, Gust J, Lim M, Majzner RG, Park JR, Partap S, Ramakrishna S, Richards R, Schultz L, Vitanza NA, Wang LD, Mackall CL, Monje M. Mahdi J, et al. Nat Med. 2023 Apr;29(4):803-810. doi: 10.1038/s41591-023-02276-w. Epub 2023 Apr 6. Nat Med. 2023. PMID: 37024595 Free PMC article. Review.
Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blind, randomised, phase 3 trial.
Heijerman HGM, McKone EF, Downey DG, Van Braeckel E, Rowe SM, Tullis E, Mall MA, Welter JJ, Ramsey BW, McKee CM, Marigowda G, Moskowitz SM, Waltz D, Sosnay PR, Simard C, Ahluwalia N, Xuan F, Zhang Y, Taylor-Cousar JL, McCoy KS; VX17-445-103 Trial Group. Heijerman HGM, et al. Lancet. 2019 Nov 23;394(10212):1940-1948. doi: 10.1016/S0140-6736(19)32597-8. Epub 2019 Oct 31. Lancet. 2019. PMID: 31679946 Free PMC article. Clinical Trial.
Intraventricular B7-H3 CAR T Cells for Diffuse Intrinsic Pontine Glioma: Preliminary First-in-Human Bioactivity and Safety.
Vitanza NA, Wilson AL, Huang W, Seidel K, Brown C, Gustafson JA, Yokoyama JK, Johnson AJ, Baxter BA, Koning RW, Reid AN, Meechan M, Biery MC, Myers C, Rawlings-Rhea SD, Albert CM, Browd SR, Hauptman JS, Lee A, Ojemann JG, Berens ME, Dun MD, Foster JB, Crotty EE, Leary SES, Cole BL, Perez FA, Wright JN, Orentas RJ, Chour T, Newell EW, Whiteaker JR, Zhao L, Paulovich AG, Pinto N, Gust J, Gardner RA, Jensen MC, Park JR. Vitanza NA, et al. Cancer Discov. 2023 Jan 9;13(1):114-131. doi: 10.1158/2159-8290.CD-22-0750. Cancer Discov. 2023. PMID: 36259971 Free PMC article.
Locoregional infusion of HER2-specific CAR T cells in children and young adults with recurrent or refractory CNS tumors: an interim analysis.
Vitanza NA, Johnson AJ, Wilson AL, Brown C, Yokoyama JK, Künkele A, Chang CA, Rawlings-Rhea S, Huang W, Seidel K, Albert CM, Pinto N, Gust J, Finn LS, Ojemann JG, Wright J, Orentas RJ, Baldwin M, Gardner RA, Jensen MC, Park JR. Vitanza NA, et al. Nat Med. 2021 Sep;27(9):1544-1552. doi: 10.1038/s41591-021-01404-8. Epub 2021 Jul 12. Nat Med. 2021. PMID: 34253928 Clinical Trial.
Update in Cystic Fibrosis 2018.
Ramsey BW, Downey GP, Goss CH. Ramsey BW, et al. Am J Respir Crit Care Med. 2019 May 15;199(10):1188-1194. doi: 10.1164/rccm.201902-0310UP. Am J Respir Crit Care Med. 2019. PMID: 30917288 Free PMC article. Review. No abstract available.
Locoregional CAR T cells for children with CNS tumors: Clinical procedure and catheter safety.
Vitanza NA, Ronsley R, Choe M, Henson C, Breedt M, Barrios-Anderson A, Wein A, Brown C, Beebe A, Kong A, Kirkey D, Lee BM, Leary SES, Crotty EE, Hoeppner C, Holtzclaw S, Wilson AL, Gustafson JA, Foster JB, Iliff JJ, Goldstein HE, Browd SR, Lee A, Ojemann JG, Pinto N, Gust J, Gardner RA, Jensen MC, Hauptman JS, Park JR. Vitanza NA, et al. Neoplasia. 2023 Feb;36:100870. doi: 10.1016/j.neo.2022.100870. Epub 2023 Jan 3. Neoplasia. 2023. PMID: 36599192 Free PMC article.
Development of a Safety Surveillance Plan for the Academic Medicine Sponsor Performing First-in-Human Cellular Therapy Clinical Trials: A Report from the Consortium for Pediatric Cellular Immunotherapy.
Adams C, Keller M, Michlitsch JG, Aguayo-Hiraldo P, Chen K, Hossain MZ, Davis A, Park JR, Verneris MR, Gardner RA. Adams C, et al. Transplant Cell Ther. 2024 May;30(5):475-487. doi: 10.1016/j.jtct.2024.02.022. Epub 2024 Mar 4. Transplant Cell Ther. 2024. PMID: 38447751 Review.
Modified Manufacturing Process Modulates CD19CAR T-cell Engraftment Fitness and Leukemia-Free Survival in Pediatric and Young Adult Subjects.
Ceppi F, Wilson AL, Annesley C, Kimmerly GR, Summers C, Brand A, Seidel K, Wu QV, Beebe A, Brown C, Mgebroff S, Lindgren C, Rawlings-Rhea SD, Huang W, Pulsipher MA, Wayne AS, Park JR, Jensen MC, Gardner RA. Ceppi F, et al. Cancer Immunol Res. 2022 Jul 1;10(7):856-870. doi: 10.1158/2326-6066.CIR-21-0501. Cancer Immunol Res. 2022. PMID: 35580141 Free PMC article. Clinical Trial.
15 results