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281. VV116 versus Nirmatrelvir-Ritonavir for Oral Treatment of Covid-19.
作者: Zhujun Cao.;Weiyi Gao.;Hong Bao.;Haiyan Feng.;Shuya Mei.;Peizhan Chen.;Yueqiu Gao.;Zhilei Cui.;Qin Zhang.;Xianmin Meng.;Honglian Gui.;Weijing Wang.;Yimei Jiang.;Zijia Song.;Yiqing Shi.;Jing Sun.;Yifei Zhang.;Qing Xie.;Yiping Xu.;Guang Ning.;Yuan Gao.;Ren Zhao.
来源: N Engl J Med. 2023年388卷5期406-417页
Nirmatrelvir-ritonavir has been authorized for emergency use by many countries for the treatment of coronavirus disease 2019 (Covid-19). However, the supply falls short of the global demand, which creates a need for more options. VV116 is an oral antiviral agent with potent activity against severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2).
282. Neutralization against BA.2.75.2, BQ.1.1, and XBB from mRNA Bivalent Booster.
作者: Meredith E Davis-Gardner.;Lilin Lai.;Bushra Wali.;Hady Samaha.;Daniel Solis.;Matthew Lee.;Andrea Porter-Morrison.;Ian T Hentenaar.;Fumiko Yamamoto.;Sucheta Godbole.;Yuan Liu.;Daniel C Douek.;Frances Eun-Hyung Lee.;Nadine Rouphael.;Alberto Moreno.;Benjamin A Pinsky.;Mehul S Suthar.
来源: N Engl J Med. 2023年388卷2期183-185页 283. Adagrasib with or without Cetuximab in Colorectal Cancer with Mutated KRAS G12C.
作者: Rona Yaeger.;Jared Weiss.;Meredith S Pelster.;Alexander I Spira.;Minal Barve.;Sai-Hong I Ou.;Ticiana A Leal.;Tanios S Bekaii-Saab.;Cloud P Paweletz.;Grace A Heavey.;James G Christensen.;Karen Velastegui.;Thian Kheoh.;Hirak Der-Torossian.;Samuel J Klempner.
来源: N Engl J Med. 2023年388卷1期44-54页
Adagrasib, an oral small-molecule inhibitor of mutant KRAS G12C protein, has shown clinical activity in pretreated patients with several tumor types, including colorectal cancer. Preclinical studies suggest that combining a KRAS G12C inhibitor with an epidermal growth factor receptor antibody could be an effective clinical strategy.
284. Sotorasib in KRAS p.G12C-Mutated Advanced Pancreatic Cancer.
作者: John H Strickler.;Hironaga Satake.;Thomas J George.;Rona Yaeger.;Antoine Hollebecque.;Ignacio Garrido-Laguna.;Martin Schuler.;Timothy F Burns.;Andrew L Coveler.;Gerald S Falchook.;Mark Vincent.;Yu Sunakawa.;Laetitia Dahan.;David Bajor.;Sun-Young Rha.;Charlotte Lemech.;Dejan Juric.;Marko Rehn.;Gataree Ngarmchamnanrith.;Pegah Jafarinasabian.;Qui Tran.;David S Hong.
来源: N Engl J Med. 2023年388卷1期33-43页
KRAS p.G12C mutation occurs in approximately 1 to 2% of pancreatic cancers. The safety and efficacy of sotorasib, a KRAS G12C inhibitor, in previously treated patients with KRAS p.G12C-mutated pancreatic cancer are unknown.
285. Lentiviral Gene Therapy for Artemis-Deficient SCID.
作者: Morton J Cowan.;Jason Yu.;Janelle Facchino.;Carol Fraser-Browne.;Ukina Sanford.;Misako Kawahara.;Jasmeen Dara.;Janel Long-Boyle.;Jess Oh.;Wendy Chan.;Shivali Chag.;Lori Broderick.;Deepak Chellapandian.;Hélène Decaluwe.;Catherine Golski.;Diana Hu.;Caroline Y Kuo.;Holly K Miller.;Aleksandra Petrovic.;Robert Currier.;Joan F Hilton.;Divya Punwani.;Christopher C Dvorak.;Harry L Malech.;R Scott McIvor.;Jennifer M Puck.
来源: N Engl J Med. 2022年387卷25期2344-2355页
The DNA-repair enzyme Artemis is essential for rearrangement of T- and B-cell receptors. Mutations in DCLRE1C, which encodes Artemis, cause Artemis-deficient severe combined immunodeficiency (ART-SCID), which is poorly responsive to allogeneic hematopoietic-cell transplantation.
287. Deep Intronic FGF14 GAA Repeat Expansion in Late-Onset Cerebellar Ataxia.
作者: David Pellerin.;Matt C Danzi.;Carlo Wilke.;Mathilde Renaud.;Sarah Fazal.;Marie-Josée Dicaire.;Carolin K Scriba.;Catherine Ashton.;Christopher Yanick.;Danique Beijer.;Adriana Rebelo.;Clarissa Rocca.;Zane Jaunmuktane.;Joshua A Sonnen.;Roxanne Larivière.;David Genís.;Laura Molina Porcel.;Karine Choquet.;Rawan Sakalla.;Sylvie Provost.;Rebecca Robertson.;Xavier Allard-Chamard.;Martine Tétreault.;Sarah J Reiling.;Sara Nagy.;Vikas Nishadham.;Meera Purushottam.;Seena Vengalil.;Mainak Bardhan.;Atchayaram Nalini.;Zhongbo Chen.;Jean Mathieu.;Rami Massie.;Colin H Chalk.;Anne-Louise Lafontaine.;François Evoy.;Marie-France Rioux.;Jiannis Ragoussis.;Kym M Boycott.;Marie-Pierre Dubé.;Antoine Duquette.;Henry Houlden.;Gianina Ravenscroft.;Nigel G Laing.;Phillipa J Lamont.;Mario A Saporta.;Rebecca Schüle.;Ludger Schöls.;Roberta La Piana.;Matthis Synofzik.;Stephan Zuchner.;Bernard Brais.
来源: N Engl J Med. 2023年388卷2期128-141页
The late-onset cerebellar ataxias (LOCAs) have largely resisted molecular diagnosis.
289. Prostate Cancer Screening with PSA and MRI Followed by Targeted Biopsy Only.
作者: Jonas Hugosson.;Marianne Månsson.;Jonas Wallström.;Ulrika Axcrona.;Sigrid V Carlsson.;Lars Egevad.;Kjell Geterud.;Ali Khatami.;Kimia Kohestani.;Carl-Gustaf Pihl.;Andreas Socratous.;Johan Stranne.;Rebecka Arnsrud Godtman.;Mikael Hellström.; .
来源: N Engl J Med. 2022年387卷23期2126-2137页
Screening for prostate cancer is burdened by a high rate of overdiagnosis. The most appropriate algorithm for population-based screening is unknown.
290. Efficacy of Antiviral Agents against Omicron Subvariants BQ.1.1 and XBB.
作者: Masaki Imai.;Mutsumi Ito.;Maki Kiso.;Seiya Yamayoshi.;Ryuta Uraki.;Shuetsu Fukushi.;Shinji Watanabe.;Tadaki Suzuki.;Ken Maeda.;Yuko Sakai-Tagawa.;Kiyoko Iwatsuki-Horimoto.;Peter J Halfmann.;Yoshihiro Kawaoka.
来源: N Engl J Med. 2023年388卷1期89-91页 292. Reemergence of Cholera in Haiti.
作者: Daniel H F Rubin.;Franz G Zingl.;Deborah R Leitner.;Ralph Ternier.;Valusnor Compere.;Samson Marseille.;Damien Slater.;Jason B Harris.;Fahima Chowdhury.;Firdausi Qadri.;Jacques Boncy.;Louise C Ivers.;Matthew K Waldor.
来源: N Engl J Med. 2022年387卷25期2387-2389页 294. Trial of Training to Reduce Driver Inattention in Teens with ADHD.
作者: Jeffery N Epstein.;Annie A Garner.;Adam W Kiefer.;James Peugh.;Leanne Tamm.;Ryan P MacPherson.;John O Simon.;Donald L Fisher.
来源: N Engl J Med. 2022年387卷22期2056-2066页
Teens with attention deficit-hyperactivity disorder (ADHD) are at increased risk for motor vehicle collisions. A computerized skills-training program to reduce long glances away from the roadway, a contributor to collision risk, may ameliorate driving risks among teens with ADHD.
295. Buprenorphine versus Methadone for Opioid Use Disorder in Pregnancy.
作者: Elizabeth A Suarez.;Krista F Huybrechts.;Loreen Straub.;Sonia Hernández-Díaz.;Hendrée E Jones.;Hilary S Connery.;Jonathan M Davis.;Kathryn J Gray.;Barry Lester.;Mishka Terplan.;Helen Mogun.;Brian T Bateman.
来源: N Engl J Med. 2022年387卷22期2033-2044页
Opioid agonist therapy is strongly recommended for pregnant persons with opioid use disorder. Buprenorphine may be associated with more favorable neonatal and maternal outcomes than methadone, but existing data are limited.
296. Neutralization of Omicron Subvariant BA.2.75 after Bivalent Vaccination.
作者: Spyros Chalkias.;Jing Feng.;Xing Chen.;Honghong Zhou.;Jean-Claude Marshall.;Bethany Girard.;Joanne E Tomassini.;Barbara J Kuter.;David C Montefiori.;Rituparna Das.
来源: N Engl J Med. 2022年387卷23期2194-2196页 297. European Haplotype of <i>Echinococcus multilocularis</i> in the United States.
作者: Louis B Polish.;Elise M O'Connell.;Thomas F E Barth.;Bruno Gottstein.;Anne Zajac.;Pamela C Gibson.;Aissatou Bah.;Megan Kirchgessner.;Marko Estrada.;M Alexis Seguin.;Roger Ramirez-Barrios.
来源: N Engl J Med. 2022年387卷20期1902-1904页 298. In Vitro Efficacy of Antiviral Agents against Omicron Subvariant BA.4.6.
作者: Emi Takashita.;Seiya Yamayoshi.;Peter Halfmann.;Nancy Wilson.;Hunter Ries.;Alex Richardson.;Max Bobholz.;William Vuyk.;Robert Maddox.;David A Baker.;Thomas C Friedrich.;David H O'Connor.;Ryuta Uraki.;Mutsumi Ito.;Yuko Sakai-Tagawa.;Eisuke Adachi.;Makoto Saito.;Michiko Koga.;Takeya Tsutsumi.;Kiyoko Iwatsuki-Horimoto.;Maki Kiso.;Hiroshi Yotsuyanagi.;Shinji Watanabe.;Hideki Hasegawa.;Masaki Imai.;Yoshihiro Kawaoka.
来源: N Engl J Med. 2022年387卷22期2094-2097页 299. IARC Perspective on Oral Cancer Prevention.
作者: Véronique Bouvard.;Suzanne T Nethan.;Deependra Singh.;Saman Warnakulasuriya.;Ravi Mehrotra.;Anil K Chaturvedi.;Tony Hsiu-Hsi Chen.;Olalekan A Ayo-Yusuf.;Prakash C Gupta.;Alexander R Kerr.;Wanninayake M Tilakaratne.;Devasena Anantharaman.;David I Conway.;Ann Gillenwater.;Newell W Johnson.;Luiz P Kowalski.;Maria E Leon.;Olena Mandrik.;Toru Nagao.;Vinayak M Prasad.;Kunnambath Ramadas.;Felipe Roitberg.;Pierre Saintigny.;Rengaswamy Sankaranarayanan.;Alan R Santos-Silva.;Dhirendra N Sinha.;Patravoot Vatanasapt.;Rosnah B Zain.;Béatrice Lauby-Secretan.
来源: N Engl J Med. 2022年387卷21期1999-2005页 300. In Utero Enzyme-Replacement Therapy for Infantile-Onset Pompe's Disease.
作者: Jennifer L Cohen.;Pranesh Chakraborty.;Karen Fung-Kee-Fung.;Marisa E Schwab.;Deeksha Bali.;Sarah P Young.;Michael H Gelb.;Hamid Khaledi.;Alicia DiBattista.;Stacey Smallshaw.;Felipe Moretti.;Derek Wong.;Catherine Lacroix.;Dina El Demellawy.;Kyle C Strickland.;Jane Lougheed.;Anita Moon-Grady.;Billie R Lianoglou.;Paul Harmatz.;Priya S Kishnani.;Tippi C MacKenzie.
来源: N Engl J Med. 2022年387卷23期2150-2158页
Patients with early-onset lysosomal storage diseases are ideal candidates for prenatal therapy because organ damage starts in utero. We report the safety and efficacy results of in utero enzyme-replacement therapy (ERT) in a fetus with CRIM (cross-reactive immunologic material)-negative infantile-onset Pompe's disease. The family history was positive for infantile-onset Pompe's disease with cardiomyopathy in two previously affected deceased siblings. After receiving in utero ERT and standard postnatal therapy, the current patient had normal cardiac and age-appropriate motor function postnatally, was meeting developmental milestones, had normal biomarker levels, and was feeding and growing well at 13 months of age.
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