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Page 1
Measuring the effect of newborn screening on survival after haematopoietic cell transplantation for severe combined immunodeficiency: a 36-year longitudinal study from the Primary Immune Deficiency Treatment Consortium.
Thakar MS, Logan BR, Puck JM, Dunn EA, Buckley RH, Cowan MJ, O'Reilly RJ, Kapoor N, Satter LF, Pai SY, Heimall J, Chandra S, Ebens CL, Chellapandian D, Williams O, Burroughs LM, Saldana BD, Rayes A, Madden LM, Chandrakasan S, Bednarski JJ 2nd, DeSantes KB, Cuvelier GDE, Teira P, Gillio AP, Eissa H, Knutsen AP, Goldman FD, Aquino VM, Shereck EB, Moore TB, Caywood EH, Lugt MTV, Rozmus J, Broglie L, Yu LC, Shah AJ, Andolina JR, Liu X, Parrott RE, Dara J, Prockop S, Martinez CA, Kapadia M, Jyonouchi SC, Sullivan KE, Bleesing JJ, Chaudhury S, Petrovic A, Keller MD, Quigg TC, Parikh S, Shenoy S, Seroogy C, Rubin T, Decaluwe H, Routes JM, Torgerson TR, Leiding JW, Pulsipher MA, Kohn DB, Griffith LM, Haddad E, Dvorak CC, Notarangelo LD. Thakar MS, et al. Among authors: goldman fd. Lancet. 2023 Jul 8;402(10396):129-140. doi: 10.1016/S0140-6736(23)00731-6. Epub 2023 Jun 20. Lancet. 2023. PMID: 37352885 Free PMC article.
Primary Immune Deficiency Treatment Consortium (PIDTC) report.
Griffith LM, Cowan MJ, Notarangelo LD, Kohn DB, Puck JM, Pai SY, Ballard B, Bauer SC, Bleesing JJ, Boyle M, Brower A, Buckley RH, van der Burg M, Burroughs LM, Candotti F, Cant AJ, Chatila T, Cunningham-Rundles C, Dinauer MC, Dvorak CC, Filipovich AH, Fleisher TA, Bobby Gaspar H, Gungor T, Haddad E, Hovermale E, Huang F, Hurley A, Hurley M, Iyengar S, Kang EM, Logan BR, Long-Boyle JR, Malech HL, McGhee SA, Modell F, Modell V, Ochs HD, O'Reilly RJ, Parkman R, Rawlings DJ, Routes JM, Shearer WT, Small TN, Smith H, Sullivan KE, Szabolcs P, Thrasher A, Torgerson TR, Veys P, Weinberg K, Zuniga-Pflucker JC; workshop participants. Griffith LM, et al. J Allergy Clin Immunol. 2014 Feb;133(2):335-47. doi: 10.1016/j.jaci.2013.07.052. Epub 2013 Oct 15. J Allergy Clin Immunol. 2014. PMID: 24139498 Free PMC article.
The diagnosis of severe combined immunodeficiency: Implementation of the PIDTC 2022 Definitions.
Dvorak CC, Haddad E, Heimall J, Dunn E, Cowan MJ, Pai SY, Kapoor N, Satter LF, Buckley RH, O'Reilly RJ, Chandra S, Bednarski JJ, Williams O, Rayes A, Moore TB, Ebens CL, Davila Saldana BJ, Petrovic A, Chellapandian D, Cuvelier GDE, Vander Lugt MT, Caywood EH, Chandrakasan S, Eissa H, Goldman FD, Shereck E, Aquino VM, Desantes KB, Madden LM, Miller HK, Yu L, Broglie L, Gillio A, Shah AJ, Knutsen AP, Andolina JP, Joshi AY, Szabolcs P, Kapadia M, Martinez CA, Parrot RE, Sullivan KE, Prockop SE, Abraham RS, Thakar MS, Leiding JW, Kohn DB, Pulsipher MA, Griffith LM, Notarangelo LD, Puck JM. Dvorak CC, et al. Among authors: goldman fd. J Allergy Clin Immunol. 2023 Feb;151(2):547-555.e5. doi: 10.1016/j.jaci.2022.10.021. Epub 2022 Nov 28. J Allergy Clin Immunol. 2023. PMID: 36456360 Free PMC article.
Liver disease and transplantation in telomere biology disorders: An international multicenter cohort.
Wang YM, Kaj-Carbaidwala B, Lane A, Agarwal S, Beier F, Bertuch A, Borovsky KA, Brennan SK, Calado RT, Catto LFB, Dufour C, Ebens CL, Fioredda F, Giri N, Gloude N, Goldman F, Hertel PM, Himes R, Keel SB, Koura DT, Kratz CP, Kulkarni S, Liou I, Nakano TA, Nastasio S, Niewisch MR, Penrice DD, Sasa GS, Savage SA, Simonetto DA, Ziegler DS, Miethke AG, Myers KC; Clinical Care Consortium for Telomere-associated Ailments (CCCTAA). Wang YM, et al. Hepatol Commun. 2024 Jun 19;8(7):e0462. doi: 10.1097/HC9.0000000000000462. eCollection 2024 Jul 1. Hepatol Commun. 2024. PMID: 38896081 Free PMC article.
Severe, life-threatening, and fatal chronic health conditions after allogeneic blood or marrow transplantation in childhood.
Holmqvist AS, Chen Y, Hageman L, Landier W, Wu J, Francisco LF, Ross ES, Balas NA, Bosworth A, Te HS, Goldman FD, Rosenthal J, Wong FL, Weisdorf DJ, Armenian SH, Bhatia S. Holmqvist AS, et al. Among authors: goldman fd. Cancer. 2023 Feb 15;129(4):624-633. doi: 10.1002/cncr.34575. Epub 2022 Dec 9. Cancer. 2023. PMID: 36484292 Free PMC article.
Wiskott-Aldrich syndrome: a study of 577 patients defines the genotype as a biomarker for disease severity and survival.
Vallée TC, Glasmacher JS, Buchner H, Arkwright PD, Behrends U, Bondarenko A, Browning MJ, Buchbinder D, Cattoni A, Chernyshova L, Ciznar P, Cole T, Czogała W, Dueckers G, Edgar JDM, Erbey F, Fasth A, Ferrua F, Formankova R, Gambineri E, Gennery AR, Goldman FD, Gonzalez-Granado LI, Heilmann C, Heiskanen-Kosma T, Juntti H, Kainulainen L, Kanegane H, Karaca NE, Kilic SS, Klein C, Kołtan S, Kondratenko I, Meyts I, Nasrullayeva GM, Notarangelo LD, Pasic S, Pellier I, Pignata C, Misbah S, Schulz A, Segundo GR, Shcherbina A, Slatter M, Sokolic R, Soler-Palacin P, Stepensky P, van Montfrans JM, Ryhänen S, Wolska-Kuśnierz B, Ziegler JB, Zhao X, Aiuti A, Ochs HD, Albert MH. Vallée TC, et al. Among authors: goldman fd. Blood. 2024 Jun 13;143(24):2504-2516. doi: 10.1182/blood.2023021411. Blood. 2024. PMID: 38579284
75 results