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Regulating Drug Prices while Increasing Innovation
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
Drug-Pricing Debate Redux — Should Cost-Effectiveness Analysis Be Used Now to Price Pharmaceuticals?
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
Revival
Joshua Schor, M.D., illustrated by Natalie Koscal, M.A., and Kim Knoper, M.A.
doi : 10.1056/NEJMp2108154
Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome
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.
Early Treatment for Covid-19 with SARS-CoV-2 Neutralizing Antibody Sotrovimab
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.
Early Convalescent Plasma for High-Risk Outpatients with Covid-19
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.
Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A
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.
Parathyroid Hormone Resistance and Autoantibodies to the PTH1 Receptor
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
Insights into Salt Handling and Blood Pressure
David H. Ellison, M.D., and Paul Welling, M.D.
doi : 10.1056/NEJMra2030212
N Engl J Med 2021; 385:1981-1993
Retinopathy in Malignant Hypertension
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
Gingival Melanoma
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
Case 35-2021: A 50-Year-Old Woman with Pain in the Left Upper Quadrant and Hypoxemia
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
The Beginning of the End of Allogeneic Transplantation for Hurler Syndrome?
Sandhya Kharbanda, M.D., and Christopher C. Dvorak, M.D.
doi : 10.1056/NEJMe2116020
N Engl J Med 2021; 385:2003-2004
Audio Interview: The Legal Basis for Covid-19 Restrictions and Mandates
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
Eliminating the “Hanger” from Hunger
J. Nicholas Betley, Ph.D.
doi : 10.1056/NEJMcibr2111841
N Engl J Med 2021; 385:2005-2007
Covid-19 Vaccination during Pregnancy and First-Trimester Miscarriage
doi : 10.1056/NEJMc2114466
N Engl J Med 2021; 385:2008-2010
Differential Kinetics of Immune Responses Elicited by Covid-19 Vaccines
doi : 10.1056/NEJMc2115596
N Engl J Med 2021; 385:2010-2012
Therapeutic Anticoagulation with Heparin in Patients with Covid-19
doi : 10.1056/NEJMc2115560
N Engl J Med 2021; 385:2013-2014
Long-Term Complications in Youth-Onset Type 2 Diabetes
doi : 10.1056/NEJMc2114053
N Engl J Med 2021; 385:2014-2016
Reframing Immune-Mediated Inflammatory Diseases
doi : 10.1056/NEJMc2114894
N Engl J Med 2021; 385:e75
