Rena M. Conti, Ph.D., Richard G. Frank, Ph.D., and Jonathan Gruber, Ph.D.
doi : 10.1056/NEJMp2113764
N Engl J Med 2021; 385:1921-1923
Peter J. Neumann, Sc.D., Joshua T. Cohen, Ph.D., and Daniel A. Ollendorf, Ph.D.
doi : 10.1056/NEJMp2113323
N Engl J Med 2021; 385:1923-1924
Joshua Schor, M.D., illustrated by Natalie Koscal, M.A., and Kim Knoper, M.A.
doi : 10.1056/NEJMp2108154
Bernhard Gentner, M.D., Francesca Tucci, M.D., Stefania Galimberti, Ph.D., Francesca Fumagalli, M.D., Maurizio De Pellegrin, M.D., Paolo Silvani, M.D., Chiara Camesasca, M.D., Silvia Pontesilli, M.D., Silvia Darin, R.N., Francesca Ciotti, Psy.D., Marina Sarzana, P.T., Giulia Consiglieri, M.D., Chiara Filisetti, M.D., Giulia Forni, B.Sc., Laura Passerini, Ph.D., Daniela Tomasoni, B.Sc., Daniela Cesana, Ph.D., Andrea Calabria, Ph.D., Giulio Spinozzi, Ph.D., Maria-Pia Cicalese, M.D., Ph.D., Valeria Calbi, M.D., Maddalena Migliavacca, M.D., Ph.D., Federica Barzaghi, M.D., Ph.D., Francesca Ferrua, M.D., Ph.D., Vera Gallo, M.D., Simona Miglietta, M.Sc., Erika Zonari, Ph.D., Patali S. Cheruku, M.D., Claudia Forni, Ph.D., Marcella Facchini, Ph.D., Ambra Corti, B.Sc., Michela Gabaldo, Pharm.D., Stefano Zancan, M.Sc., Serena Gasperini, M.D., Attilio Rovelli, M.D., Jaap-Jan Boelens, M.D., Ph.D., Simon A. Jones, M.D., Robert Wynn, M.D., Cristina Baldoli, M.D., Eugenio Montini, Ph.D., Silvia Gregori, Ph.D., Fabio Ciceri, M.D., Maria G. Valsecchi, Ph.D., Giancarlo la Marca, Pharm.Sc., Rossella Parini, M.D., Luigi Naldini, M.D., Ph.D., Alessandro Aiuti, M.D., Ph.D., and Maria-Ester Bernardo, M.D., Ph.D. for the MPSI Study Group*
doi : 10.1056/NEJMoa2106596
N Engl J Med 2021; 385:1929-1940
Allogeneic hematopoietic stem-cell transplantation is the standard of care for Hurler syndrome (mucopolysaccharidosis type I, Hurler variant [MPSIH]). However, this treatment is only partially curative and is associated with complications.
Anil Gupta, M.D., Yaneicy Gonzalez-Rojas, M.D., Erick Juarez, M.D., Manuel Crespo Casal, M.D., Jaynier Moya, M.D., Diego R. Falci, M.D., Ph.D., Elias Sarkis, M.D., Joel Solis, M.D., Hanzhe Zheng, Ph.D., Nicola Scott, M.Sc., Andrea L. Cathcart, Ph.D., Christy M. Hebner, Ph.D., Jennifer Sager, Ph.D., Erik Mogalian, Pharm.D., Ph.D., Craig Tipple, M.B., B.S., Ph.D., Amanda Peppercorn, M.D., Elizabeth Alexander, M.D., Phillip S. Pang, M.D., Ph.D., Almena Free, M.D., Cynthia Brinson, M.D., Melissa Aldinger, Pharm.D., and Adrienne E. Shapiro, M.D., Ph.D. for the COMET-ICE Investigators*
doi : 10.1056/NEJMoa2107934
N Engl J Med 2021; 385:1941-1950
Coronavirus disease 2019 (Covid-19) disproportionately results in hospitalization or death in older patients and those with underlying conditions. Sotrovimab is a pan-sarbecovirus monoclonal antibody that was designed to prevent progression of Covid-19 in high-risk patients early in the course of disease.
Frederick K. Korley, M.D., Ph.D., Valerie Durkalski-Mauldin, Ph.D., Sharon D. Yeatts, Ph.D., Kevin Schulman, M.D., Robertson D. Davenport, M.D., Larry J. Dumont, Ph.D., Nahed El Kassar, M.D., Ph.D., Lydia D. Foster, M.S., Jennifer M. Hah, M.D., Siddartha Jaiswal, M.D., Ph.D., Alesia Kaplan, M.D., Ezekiel Lowell, B.S., John F. McDyer, M.D., James Quinn, M.D., Darrell J. Triulzi, M.D., Carol Van Huysen, B.S., Valerie L.W. Stevenson, B.A.S., R.R.T., Kabir Yadav, M.D.C.M., Christopher W. Jones, M.D., Bory Kea, M.D., Aaron Burnett, M.D., Joshua C. Reynolds, M.D., Colin F. Greineder, M.D., Ph.D., Nathan L. Haas, M.D., David G. Beiser, M.D., Robert Silbergleit, M.D., William Barsan, M.D., and Clifton W. Callaway, M.D., Ph.D. for the SIREN-C3PO Investigators*
doi : 10.1056/NEJMoa2103784
N Engl J Med 2021; 385:1951-1960
Early administration of convalescent plasma obtained from blood donors who have recovered from coronavirus disease 2019 (Covid-19) may prevent disease progression in acutely ill, high-risk patients with Covid-19.
Lindsey A. George, M.D., Paul E. Monahan, M.D., M. Elaine Eyster, M.D., Spencer K. Sullivan, M.D., Margaret V. Ragni, M.D., M.P.H., Stacy E. Croteau, M.D., M.M.S., John E.J. Rasko, M.B., B.S., Ph.D., Michael Recht, M.D., Ph.D., Benjamin J. Samelson-Jones, M.D., Ph.D., Amy MacDougall, B.S.N., Kristen Jaworski, M.S.N., F.N.P., Robert Noble, Ph.D., Marla Curran, Dr.P.H., Klaudia Kuranda, Ph.D., Federico Mingozzi, Ph.D., Tiffany Chang, M.D., Kathleen Z. Reape, M.D., Xavier M. Anguela, Ph.D., and Katherine A. High, M.D.
doi : 10.1056/NEJMoa2104205
N Engl J Med 2021; 385:1961-1973
The goal of gene therapy for patients with hemophilia A is to safely impart long-term stable factor VIII expression that predictably ameliorates bleeding with the use of the lowest possible vector dose.
Adel Mandl, M.D., Ph.D., Peter D. Burbelo, Ph.D., Giovanni Di Pasquale, Ph.D., You Sher Tay, M.D., James Welch, M.G.C., Michail S. Lionakis, M.D., Sergio D. Rosenzweig, M.D., Ph.D., Meryl A. Waldman, M.D., Blake M. Warner, D.D.S., Ph.D., M.P.H., Brian Walitt, M.D., M.P.H., Michael T. Collins, M.D., James E. Balow, M.D., John A. Chiorini, Ph.D., William F. Simonds, M.D., Sunita K. Agarwal, Ph.D., Jenny E. Blau, M.D., and Lee S. Weinstein, M.D.
doi : 10.1056/NEJMoa2109409
N Engl J Med 2021; 385:1974-1980
David H. Ellison, M.D., and Paul Welling, M.D.
doi : 10.1056/NEJMra2030212
N Engl J Med 2021; 385:1981-1993
Federica Poli, M.B., Ch.B., and Imran H. Yusuf, M.B., Ch.B.
doi : 10.1056/NEJMicm2109500
N Engl J Med 2021; 385:1994
Elena M. Varoni, Ph.D., D.D.S., and Sem Decani, D.D.S.
doi : 10.1056/NEJMicm2107766
N Engl J Med 2021; 385:e73
Margaret M. Chapman, M.D., Victorine V. Muse, M.D., James E. Mojica, M.D., and Melis N. Anahtar, M.D., Ph.D.
doi : 10.1056/NEJMcpc2107356
N Engl J Med 2021; 385:1995-2001
Sandhya Kharbanda, M.D., and Christopher C. Dvorak, M.D.
doi : 10.1056/NEJMe2116020
N Engl J Med 2021; 385:2003-2004
Eric J. Rubin, M.D., Ph.D., Lindsey R. Baden, M.D., Michelle M. Mello, J.D., Ph.D., and Stephen Morrissey, Ph.D.
doi : 10.1056/NEJMe2118197
N Engl J Med 2021; 385:e88
J. Nicholas Betley, Ph.D.
doi : 10.1056/NEJMcibr2111841
N Engl J Med 2021; 385:2005-2007
doi : 10.1056/NEJMc2114466
N Engl J Med 2021; 385:2008-2010
doi : 10.1056/NEJMc2115596
N Engl J Med 2021; 385:2010-2012
doi : 10.1056/NEJMc2115560
N Engl J Med 2021; 385:2013-2014
doi : 10.1056/NEJMc2114053
N Engl J Med 2021; 385:2014-2016
doi : 10.1056/NEJMc2114894
N Engl J Med 2021; 385:e75
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