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of 102
pro vyhledávání: ''
Autor:
Donald B. Kohn, Claire Booth, Kit L. Shaw, Jinhua Xu-Bayford, Elizabeth Garabedian, Valentina Trevisan, Denise A. Carbonaro-Sarracino, Kajal Soni, Dayna Terrazas, Katie Snell, Alan Ikeda, Diego Leon-Rico, Theodore B. Moore, Karen F. Buckland, Ami J. Shah, Kimberly C. Gilmour, Satiro De Oliveira, Christine Rivat, Gay M. Crooks, Natalia Izotova, John Tse, Stuart Adams, Sally Shupien, Hilory Ricketts, Alejandra Davila, Chilenwa Uzowuru, Amalia Icreverzi, Provaboti Barman, Beatriz Campo Fernandez, Roger P. Hollis, Maritess Coronel, Allen Yu, Krista M. Chun, Christian E. Casas, Ruixue Zhang, Serena Arduini, Frances Lynn, Mahesh Kudari, Andrea Spezzi, Marco Zahn, Rene Heimke, Ivan Labik, Roberta Parrott, Rebecca H. Buckley, Lilith Reeves, Kenneth Cornetta, Robert Sokolic, Michael Hershfield, Manfred Schmidt, Fabio Candotti, Harry L. Malech, Adrian J. Thrasher, H. Bobby Gaspar
Publikováno v:
N Engl J Med
The New England journal of medicine, vol 384, iss 21
The New England journal of medicine, vol 384, iss 21
BACKGROUND: Severe combined immunodeficiency due to adenosine deaminase (ADA) deficiency (ADA-SCID) is a rare and life-threatening primary immunodeficiency. METHODS: We treated 50 patients with ADA-SCID (30 in the United States and 20 in the United K
Autor:
Michael E. Wechsler, Elliot Israel, Primal Kaur, Janet M. Griffiths, Christopher E. Brightling, Arnaud Bourdin, Andrew Menzies-gow, Gene L. Colice, Geoffrey Chupp, Gun Almqvist, Jonathan Corren, Karin Bowen, Sandhia Ponnarambil, Åsa Hellqvist
Publikováno v:
New England Journal of Medicine
New England Journal of Medicine, Massachusetts Medical Society, 2021, 384 (19), pp.1800-1809. ⟨10.1056/NEJMoa2034975⟩
New England Journal of Medicine, Massachusetts Medical Society, 2021, 384 (19), pp.1800-1809. ⟨10.1056/NEJMoa2034975⟩
International audience; BACKGROUND: Tezepelumab is a human monoclonal antibody that blocks thymic stromal lymphopoietin, an epithelial-cell–derived cytokine implicated in the pathogenesis of asthma. The efficacy and safety of tezepelumab in patient
Autor:
Kevan C, Herold, Brian N, Bundy, S Alice, Long, Jeffrey A, Bluestone, Linda A, DiMeglio, Matthew J, Dufort, Stephen E, Gitelman, Peter A, Gottlieb, Jeffrey P, Krischer, Peter S, Linsley, Jennifer B, Marks, Wayne, Moore, Antoinette, Moran, Henry, Rodriguez, William E, Russell, Desmond, Schatz, Jay S, Skyler, Eva, Tsalikian, Diane K, Wherrett, Anette-Gabriele, Ziegler, Carla J, Greenbaum, Stuart, Weinzimer
Publikováno v:
The New England journal of medicine, vol 381, iss 7
BackgroundType 1 diabetes is a chronic autoimmune disease that leads to destruction of insulin-producing beta cells and dependence on exogenous insulin for survival. Some interventions have delayed the loss of insulin production in patients with type
Autor:
Ostergaard, L., Vesikari, T., Absalon, J., Beeslaar, J., Ward, B. J., Senders, S., Eiden, J. J., Jansen, K. U., Anderson, A. S., York, L. J., Jones, T. R., Harris, S. L., O'Neill, R., Radley, D., Maansson, R., Pregaldien, J. -L., Ginis, J., Staerke, N. B., Perez, J. L., Belle-Isle, J, Elfassi, E, Fredette, P, Garfield, H, Girard, G, Lachance, P, Adamkova, E, Bartonova, E, Drazan, D, Dvorakova, J, Kosina, P, Kyjonkova, A, Ruzkova, R, Vitousova, E, Ahonen, A, Forsten, A, Karppa, T, Kokko, S, Lagerstrom-Tirri, Pm, Simila, Jk, Adelt, T, Behre, U, Schwarz, Tf, Castiglia, P, Esposito, S, Ferrera, G, Icardi, G, Brzostek, J, Hasiec, B, Konior, R, Pejcz, J, Szymanski, H, Witor, A, Faust, Sn, Finn, Ah, Heath, Pt, Pollard, Aj, Altamirano, Dd, Ashley CT Jr, Bader, Gf, Bauer GH Jr, Block SL Jr, Brandon, Dm, Davis, Mg, Devalle, O, Egelhof, Rh, Essink, Bj, Fouch, Bb, Fox, Bp, Franklin, Er, Garscadden, Ag, Goswami, Up, Gregory, Dm, Helman, Ll, Houchin, Vg, Howard, Ce, Johnson, Ad, Johnston WH Jr, Jordan, Ca, Kimmel, Ma, Klein, Tr, Krilov, Lr, Labarbera, Ap, Labuda JM II, Latiolais, Tg, Lello, Lg, Lewis, Dh, Ley, Ja, London, Al, Martin, Ms, Mcguire, Mr, Mosteller, Vc, Naccarato, Tr, Nassim, Cg, Rey, Mr, Robbins, Ra, Rouse, Kg, Schear, Mj, Senders, Sd, Shepard, Js, Simpson, Mw, Slandzicki, Aj, Slechta, Sb, Tetrick, Ll, Varman, M, Wadsworth LT III, Ware, Db, White, Jh, Wisman PP Jr, Blouin, F, Dionne, M, Dzongowski, P, Heaton, Kj, Langley, Jm, O'Mahony, Mfj, Powell, Cn, Ward, Bj, Ostergaard, Lj, Haapaniemi, Tl, Paassilta, M, Volanen, Ik, Lepich, T, Smukalska, E, Tarczon, I, Tetiurka, Bm, Domingo, Jd, Morato, Av, Riera, Mt, Sanchez, Ca, Torrell, Jmr, Blumenau, J, Campbell, Ng, Cervantes, Ja, Douglas, Wg, Ensz, Dj, Ervin, Je, Fiel, Tc, Fragoso, Vg, Fried, Dl, Gleason, Gp, Green, Sl, Haggag, Az, Johnson, Ct, Khaira, Rs, Kirstein, Jl, Kravitz, Ae, Lederman, Sn, Marcadis, I, Miller, Ve, Moretti, Jm, Pragalos, Aa, Puopolo, Ad, Rubino, J, Seiden, Dj, Sharp, Sc, Sheldon, Ea, Shockey, Gr, Smith, Wb, Stringer, Jc, Strout, Cb, Studdard, He, Tresser, Njl.
Publikováno v:
Ostergaard, L, Vesikari, T, Absalon, J, Beeslaar, J, Ward, B J, Senders, S, Eiden, J J, Jansen, K U, Anderson, A S, York, L J, Jones, T R, Harris, S L, O'Neill, R, Radley, D, Maansson, R, Prégaldien, J-L, Ginis, J, Staerke, N B, Perez, J L & B1971009 and B1971016 Trial Investigators 2017, ' A Bivalent Meningococcal B Vaccine in Adolescents and Young Adults ', The New England Journal of Medicine, vol. 377, no. 24, pp. 2349-2362 . https://doi.org/10.1056/NEJMoa1614474
BACKGROUND: MenB-FHbp is a licensed meningococcal B vaccine targeting factor H-binding protein. Two phase 3 studies assessed the safety of the vaccine and its immunogenicity against diverse strains of group B meningococcus.METHODS: We randomly assign
Autor:
Masashi Taki, Katsuyuki Fukutake, Koichiro Yoneyama, Tetsuji Sato, Midori Shima, Naoki Fukazawa, Hideji Hanabusa, Hiroki Yoshida, Tadashi Matsushita, Keiji Nogami
Publikováno v:
New England Journal of Medicine. 374:2044-2053
In patients with severe hemophilia A, standard treatment is regular prophylactic and episodic intravenous infusions of factor VIII. However, these treatments are burdensome, especially for children, and may lead to the formation of anti-factor VIII a
Autor:
Hanna Mandel, Dror Mevorach, Vered Yahalom, Adi Mory, David Azoulay, Omer Weissbrod, Hagit N. Baris, Yaniv Zohar, Lilach Finkel, Adi Tabib, Ron Shaoul, Sarit Peleg, Alina Kurolap, Lilach Bonstein, Orly Eshach-Adiv, Daniella Magen, Elizabeth E. Half, Tamar Paperna, Danit Oz-Levi, Judith Chezar, Tova Hershkovitz, Dan Geiger
Publikováno v:
New England Journal of Medicine. 377:87-89
CD55 (complement decay-accelerating factor) inhibits the alternative and classical arms of the complement pathway. Three patients with protein-losing enteropathy and a genetic variant predicted to result in loss of function of CD55 had a response to
Autor:
Mohammed A. Sadat, Susan Moir, Tae-Wook Chun, Paolo Lusso, Gerardo Kaplan, Lynne Wolfe, Matthew J. Memoli, Miao He, Hugo Vega, Leo J.Y. Kim, Yan Huang, Nadia Hussein, Elma Nievas, Raquel Mitchell, Mary Garofalo, Aaron Louie, Derek C. Ireland, Claire Grunes, Raffaello Cimbro, Vyomesh Patel, Genevieve Holzapfel, Daniel Salahuddin, Tyler Bristol, David Adams, Beatriz E. Marciano, Madhuri Hegde, Yuxing Li, Katherine R. Calvo, Jennifer Stoddard, J. Shawn Justement, Jerome Jacques, Debra A. Long Priel, Danielle Murray, Peter Sun, Douglas B. Kuhns, Cornelius F. Boerkoel, John A. Chiorini, Giovanni Di Pasquale, Daniela Verthelyi, Sergio D. Rosenzweig
Publikováno v:
New England Journal of Medicine. 370:1615-1625
Genetic defects in MOGS, the gene encoding mannosyl-oligosaccharide glucosidase (the first enzyme in the processing pathway of N-linked oligosaccharide), cause the rare congenital disorder of glycosylation type IIb (CDG-IIb), also known as MOGS-CDG.
Autor:
Eryu Wang, Scott C. Weaver, Julio Cisneros, Ivan Abadía, Yamilka Díaz, Dora Estripeaut, Sandra López-Vergès, Hector G. Cedeño, Nestor Sosa, Andrew D. Haddow, Amy Y. Vittor, Davis Beltran, Robert B. Tesh, Alex O. Martínez-Torres, Amelia P.A. Travassos Da Rosa, Jean-Paul Carrera, Elizabeth Castaño, Carmen Báez, Naomi L. Forrester, Humberto Hernandez
Publikováno v:
New England Journal of Medicine. 369:732-744
The eastern equine encephalitis (EEE) and Venezuelan equine encephalitis (VEE) viruses are pathogens that infect humans and horses in the Americas. Outbreaks of neurologic disease in humans and horses were reported in Panama from May through early Au
Autor:
Janice Canvin, Sunil Agarwal, Thomas B. Casale, Marcus Maurer, Ana Giménez-Arnau, Sarbjit S. Saini, Ramona Doyle, Allen P. Kaplan, Hsin-Ju Hsieh, Karin Rosén, Clive Grattan
Publikováno v:
New England Journal of Medicine. 368:924-935
Many patients with chronic idiopathic urticaria (also called chronic spontaneous urticaria) do not have a response to therapy with H-antihistamines, even at high doses. In phase 2 trials, omalizumab, an anti-IgE monoclonal antibody [corrected] that t
Autor:
Gouw, S.C., Bom, J.G. van der, Ljung, R., Escuriola, C., Cid, A.R., Claeyssens-Donadel, S., Geet, C. van, Kenet, G., Makipernaa, A., Molinari, A.C., Muntean, W., Kobelt, R., Rivard, G., Santagostino, E., Thomas, A., Berg, H.M. van den, PedNet RODIN Study Grp
Publikováno v:
New England journal of medicine, 368(3), 231-239. Massachussetts Medical Society
For previously untreated children with severe hemophilia A, it is unclear whether the type of factor VIII product administered and switching among products are associated with the development of clinically relevant inhibitory antibodies (inhibitor de