New England Journal of Medicine




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سفارش

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

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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

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Revival

Joshua Schor, M.D., illustrated by Natalie Koscal, M.A., and Kim Knoper, M.A.

doi : 10.1056/NEJMp2108154

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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.

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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.

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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.

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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.

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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

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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

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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

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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

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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

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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

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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

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Eliminating the “Hanger” from Hunger

J. Nicholas Betley, Ph.D.

doi : 10.1056/NEJMcibr2111841

N Engl J Med 2021; 385:2005-2007

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Covid-19 Vaccination during Pregnancy and First-Trimester Miscarriage

doi : 10.1056/NEJMc2114466

N Engl J Med 2021; 385:2008-2010

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Differential Kinetics of Immune Responses Elicited by Covid-19 Vaccines

doi : 10.1056/NEJMc2115596

N Engl J Med 2021; 385:2010-2012

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Therapeutic Anticoagulation with Heparin in Patients with Covid-19

doi : 10.1056/NEJMc2115560

N Engl J Med 2021; 385:2013-2014

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Long-Term Complications in Youth-Onset Type 2 Diabetes

doi : 10.1056/NEJMc2114053

N Engl J Med 2021; 385:2014-2016

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Reframing Immune-Mediated Inflammatory Diseases

doi : 10.1056/NEJMc2114894

N Engl J Med 2021; 385:e75

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Mobility Assessment in Older Adults

doi : 10.1056/NEJMc2115294

N Engl J Med 2021; 385:e76

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