当前位置: 首页 >> 检索结果
82. Recombinant ADAMTS13 in Congenital Thrombotic Thrombocytopenic Purpura.
作者: Marie Scully.;Ana Antun.;Spero R Cataland.;Paul Coppo.;Claire Dossier.;Nathalie Biebuyck.;Wolf-Achim Hassenpflug.;Karim Kentouche.;Paul Knöbl.;Johanna A Kremer Hovinga.;M Fernanda López-Fernández.;Masanori Matsumoto.;Thomas L Ortel.;Jerzy Windyga.;Indranil Bhattacharya.;Michael Cronin.;Hong Li.;Björn Mellgård.;Munjal Patel.;Parth Patwari.;Shan Xiao.;Pinghai Zhang.;Linda T Wang.; .
来源: N Engl J Med. 2024年390卷17期1584-1596页
Congenital thrombotic thrombocytopenic purpura (TTP) results from severe hereditary deficiency of ADAMTS13. The efficacy and safety of recombinant ADAMTS13 and standard therapy (plasma-derived products) administered as routine prophylaxis or on-demand treatment in patients with congenital TTP is not known.
83. Subcutaneous Administration of a Monoclonal Antibody to Prevent Malaria.
作者: Kassoum Kayentao.;Aissata Ongoiba.;Anne C Preston.;Sara A Healy.;Zonghui Hu.;Jeff Skinner.;Safiatou Doumbo.;Jing Wang.;Hamidou Cisse.;Didier Doumtabe.;Abdrahamane Traore.;Hamadi Traore.;Adama Djiguiba.;Shanping Li.;Mary E Peterson.;Shinyi Telscher.;Azza H Idris.;William C Adams.;Adrian B McDermott.;Sandeep Narpala.;Bob C Lin.;Leonid Serebryannyy.;Somia P Hickman.;Andrew J McDougal.;Sandra Vazquez.;Matthew Reiber.;Judy A Stein.;Jason G Gall.;Kevin Carlton.;Philipp Schwabl.;Siriman Traore.;Mamadou Keita.;Amatigué Zéguimé.;Adama Ouattara.;M'Bouye Doucoure.;Amagana Dolo.;Sean C Murphy.;Daniel E Neafsey.;Silvia Portugal.;Abdoulaye Djimdé.;Boubacar Traore.;Robert A Seder.;Peter D Crompton.; .
来源: N Engl J Med. 2024年390卷17期1549-1559页
Subcutaneous administration of the monoclonal antibody L9LS protected adults against controlled Plasmodium falciparum infection in a phase 1 trial. Whether a monoclonal antibody administered subcutaneously can protect children from P. falciparum infection in a region where this organism is endemic is unclear.
92. Pragmatic Trial of Hospitalization Rate in Chronic Kidney Disease.
作者: Miguel A Vazquez.;George Oliver.;Ruben Amarasingham.;Venkatraghavan Sundaram.;Kevin Chan.;Chul Ahn.;Song Zhang.;Perry Bickel.;Samir M Parikh.;Barbara Wells.;R Tyler Miller.;Susan Hedayati.;Jeffrey Hastings.;Adeola Jaiyeola.;Tuan-Minh Nguyen.;Brett Moran.;Noel Santini.;Blake Barker.;Ferdinand Velasco.;Lynn Myers.;Thomas P Meehan.;Chester Fox.;Robert D Toto.; .
来源: N Engl J Med. 2024年390卷13期1196-1206页
Despite the availability of effective therapies for patients with chronic kidney disease, type 2 diabetes, and hypertension (the kidney-dysfunction triad), the results of large-scale trials examining the implementation of guideline-directed therapy to reduce the risk of death and complications in this population are lacking.
93. Nirmatrelvir for Vaccinated or Unvaccinated Adult Outpatients with Covid-19.
作者: Jennifer Hammond.;Robert J Fountaine.;Carla Yunis.;Dona Fleishaker.;Mary Almas.;Weihang Bao.;Wayne Wisemandle.;Mary Lynn Baniecki.;Victoria M Hendrick.;Veselin Kalfov.;J Abraham Simón-Campos.;Rienk Pypstra.;James M Rusnak.
来源: N Engl J Med. 2024年390卷13期1186-1195页
Nirmatrelvir in combination with ritonavir is an antiviral treatment for mild-to-moderate coronavirus disease 2019 (Covid-19). The efficacy of this treatment in patients who are at standard risk for severe Covid-19 or who are fully vaccinated and have at least one risk factor for severe Covid-19 has not been established.
95. Intrathecal Gene Therapy for Giant Axonal Neuropathy.
作者: Diana X Bharucha-Goebel.;Joshua J Todd.;Dimah Saade.;Gina Norato.;Minal Jain.;Tanya Lehky.;Rachel M Bailey.;Jessica A Chichester.;Roberto Calcedo.;Diane Armao.;A Reghan Foley.;Payam Mohassel.;Eshetu Tesfaye.;Bradley P Carlin.;Beth Seremula.;Melissa Waite.;Wadih M Zein.;Laryssa A Huryn.;Thomas O Crawford.;Charlotte J Sumner.;Ahmet Hoke.;John D Heiss.;Lawrence Charnas.;Jody E Hooper.;Thomas W Bouldin.;Elizabeth M Kang.;Denis Rybin.;Steven J Gray.;Carsten G Bönnemann.; .
来源: N Engl J Med. 2024年390卷12期1092-1104页
Giant axonal neuropathy is a rare, autosomal recessive, pediatric, polysymptomatic, neurodegenerative disorder caused by biallelic loss-of-function variants in GAN, the gene encoding gigaxonin.
96. Risk of Autism after Prenatal Topiramate, Valproate, or Lamotrigine Exposure.
作者: Sonia Hernández-Díaz.;Loreen Straub.;Brian T Bateman.;Yanmin Zhu.;Helen Mogun.;Katherine L Wisner.;Kathryn J Gray.;Barry Lester.;Christopher J McDougle.;Elyse DiCesare.;Page B Pennell.;Krista F Huybrechts.
来源: N Engl J Med. 2024年390卷12期1069-1079页
Maternal use of valproate during pregnancy has been associated with an increased risk of neurodevelopmental disorders in children. Although most studies of other antiseizure medications have not shown increased risks of these disorders, there are limited and conflicting data regarding the risk of autism spectrum disorder associated with maternal topiramate use.
98. Intraventricular CARv3-TEAM-E T Cells in Recurrent Glioblastoma.
作者: Bryan D Choi.;Elizabeth R Gerstner.;Matthew J Frigault.;Mark B Leick.;Christopher W Mount.;Leonora Balaj.;Sarah Nikiforow.;Bob S Carter.;William T Curry.;Kathleen Gallagher.;Marcela V Maus.
来源: N Engl J Med. 2024年390卷14期1290-1298页
In this first-in-human, investigator-initiated, open-label study, three participants with recurrent glioblastoma were treated with CARv3-TEAM-E T cells, which are chimeric antigen receptor (CAR) T cells engineered to target the epidermal growth factor receptor (EGFR) variant III tumor-specific antigen, as well as the wild-type EGFR protein, through secretion of a T-cell-engaging antibody molecule (TEAM). Treatment with CARv3-TEAM-E T cells did not result in adverse events greater than grade 3 or dose-limiting toxic effects. Radiographic tumor regression was dramatic and rapid, occurring within days after receipt of a single intraventricular infusion, but the responses were transient in two of the three participants. (Funded by Gateway for Cancer Research and others; INCIPIENT ClinicalTrials.gov number, NCT05660369.).
99. Microplastics and Nanoplastics in Atheromas and Cardiovascular Events.
作者: Raffaele Marfella.;Francesco Prattichizzo.;Celestino Sardu.;Gianluca Fulgenzi.;Laura Graciotti.;Tatiana Spadoni.;Nunzia D'Onofrio.;Lucia Scisciola.;Rosalba La Grotta.;Chiara Frigé.;Valeria Pellegrini.;Maurizio Municinò.;Mario Siniscalchi.;Fabio Spinetti.;Gennaro Vigliotti.;Carmine Vecchione.;Albino Carrizzo.;Giulio Accarino.;Antonio Squillante.;Giuseppe Spaziano.;Davida Mirra.;Renata Esposito.;Simona Altieri.;Giovanni Falco.;Angelo Fenti.;Simona Galoppo.;Silvana Canzano.;Ferdinando C Sasso.;Giulia Matacchione.;Fabiola Olivieri.;Franca Ferraraccio.;Iacopo Panarese.;Pasquale Paolisso.;Emanuele Barbato.;Carmine Lubritto.;Maria L Balestrieri.;Ciro Mauro.;Augusto E Caballero.;Sanjay Rajagopalan.;Antonio Ceriello.;Bruno D'Agostino.;Pasquale Iovino.;Giuseppe Paolisso.
来源: N Engl J Med. 2024年390卷10期900-910页
Microplastics and nanoplastics (MNPs) are emerging as a potential risk factor for cardiovascular disease in preclinical studies. Direct evidence that this risk extends to humans is lacking.
|