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683. Mass Administration of Azithromycin to Infants in Mali to Reduce Mortality.
作者: Fadima Cheick Haidara.;Laura Adubra.;Mahamadou Abdou.;Dagmar Alber.;Ulla Ashorn.;Yin Bun Cheung.;Elaine Cloutman-Green.;Mamadou Diallo.;Camilla Ducker.;Yue-Mei Fan.;Gwydion Gruffudd.;Lotta Hallamaa.;Tiia Haapaniemi.;Rikhard Ihamuotila.;Jane Juma.;Nigel Klein.;Juho Luoma.;Owen Martell.;Akshaya Murugesan.;Collins Okello.;Oumar Samaké.;Cheick Amadou Tidiane Traore.;Taru Vehmasto.;Kaisa Ylikruuvi.;Samba Sow.;Per Ashorn.
来源: N Engl J Med. 2025年393卷15期1498-1508页
Mass administration of azithromycin to children 1 to 59 months of age has been shown to reduce mortality among infants and children in this age group in some areas of sub-Saharan Africa. The largest effects have appeared to be among infants younger than 12 months of age, within 3 months after treatment; this observation motivated the design of the current trial.
684. Long-Term Safety and Efficacy of Gene Therapy for Adenosine Deaminase Deficiency.
作者: Claire Booth.;Katelyn Masiuk.;Konstantinos Vazouras.;Augustine Fernandes.;Jinhua Xu-Bayford.;Beatriz Campo Fernandez.;Sohini Roy.;Beatrice Curio-Penny.;Jordyn Arnold.;Dayna Terrazas.;Jack Reid.;Kimberly C Gilmour.;Stuart Adams.;Elena Alvarez Mediavilla.;Lana Mhaldien.;Grainne O'Toole.;Rima Ahmed.;Elizabeth Garabedian.;Harry Malech.;Suk See De Ravin.;Theodore B Moore.;Satiro De Oliveira.;Danilo Pellin.;Tsai-Yu Lin.;Thao Thi Dang.;Kenneth Cornetta.;Michael S Hershfield.;Havinder Hara.;Adrian J Thrasher.;H Bobby Gaspar.;Donald B Kohn.
来源: N Engl J Med. 2025年393卷15期1486-1497页
Severe combined immunodeficiency (SCID) due to adenosine deaminase (ADA) deficiency (ADA-SCID) is a life-threatening inborn error of immunity for which lentiviral gene therapy has been investigated in clinical trials.
685. A Phase 3 Trial of Telitacicept for Systemic Lupus Erythematosus.
作者: Ronald F van Vollenhoven.;Li Wang.;Joan T Merrill.;Yi Liu.;Chunde Bao.;Fen Li.;Jiankang Hu.;Chenghui Huang.;Jianhong Zhao.;Cibo Huang.;Hanyou Mo.;Wei Wei.;Fu'ai Lu.;Jingyang Li.;Dongbao Zhao.;Wenxiang Wang.;Lin Li.;Qing Zuraw.;Xiaofei Wang.;Xuebin Wang.;Jianmin Fang.;Fengchun Zhang.; .
来源: N Engl J Med. 2025年393卷15期1475-1485页
Telitacicept, a new dual inhibitor of the cytokines B-lymphocyte stimulator (BLyS) and APRIL (a proliferation-inducing ligand), showed efficacy in adults with active systemic lupus erythematosus (SLE) in a phase 2b trial when added to standard therapy.
686. Sentinel-Lymph-Node Biopsy Alone or with Lymphadenectomy in Cervical Cancer.
作者: Hua Tu.;He Huang.;Yanfang Li.;Xiaojun Chen.;Chunyan Wang.;Min Zheng.;Yanna Zhang.;Weidong Zhao.;Yanling Feng.;Ting Wan.;Yongwen Huang.;Aijun Yu.;Weiguo Lu.;Jing Xiao.;Weiwei Shan.;Ping Zhang.;Changkun Zhu.;Danbo Wang.;Hu Zhou.;Jibin Li.;Beihua Kong.;Weiwei Feng.;Xipeng Wang.;Rongzhen Luo.;Xin Huang.;Jundong Li.;Zejian Lin.;Shuzhong Yao.;Jihong Liu.; .
来源: N Engl J Med. 2025年393卷15期1463-1474页
Limited data are available on survival outcomes after sentinel-lymph-node biopsy alone as compared with lymphadenectomy in cervical cancer.
688. Toward Same-Day Genome Sequencing in the Critical Care Setting.
作者: Monica H Wojcik.;Katie Larkin.;Michelle Cipicchio.;Austin Doupnik.;Chen Zhao.;Cynthia Cech.;Douglas Lopez.;Jagadeeswaran Chandrasekar.;Joanne Leadbetter.;John Mannion.;Kendall Berg.;Mahdi Golkaram.;McKenna Osentowski.;Megan Freer.;Taylor Lehmann.;Won-Mean Lee.;Emily Ormbrek.;Marc J Prindle.;Melud Nabavi.;Amal Chaturvedi.;Chuck Seberino.;Daniel N Baker.;Cara Williams.;Diana Toledo.;Edyta Malolepsza.;Mark Fleharty.;Andrea Oza.;Sophie Low.;Alan H Beggs.;Casie A Genetti.;Gwendolyn Strickland.;Katherine N Anderson.;Wendy K Chung.;Heidi L Rehm.;Sean Hofherr.;Mark Kokoris.;Niall Lennon.; .
来源: N Engl J Med. 2025年393卷20期2063-2065页 691. DB-OTO Gene Therapy for Inherited Deafness.
作者: Vassili Valayannopoulos.;Manohar Bance.;Daniela S Carvalho.;John H Greinwald.;Steven A Harvey.;Akira Ishiyama.;Evie C Landry.;Hubert Löwenheim.;Lawrence R Lustig.;Manuel Manrique.;Robert Nash.;Rubén Polo.;Cedric V Pritchett.;Jay T Rubinstein.;A Eliot Shearer.;Ignacio Del Castillo.;Jeffery J Anderson.;C Eduardo Corrales.;Tera M Quigley.;William J Riggs.;Peter Weber.;Gary Wilson.;Susan C Irvin.;Hazem E Hassan.;Yanping Chen.;Rong Liu.;Meghan C Drummond.;Leah R Sabin.;Bret J Musser.;George D Yancopoulos.;Christos A Kyratsous.;Gary A Herman.;Aris Baras.;Jonathon P Whitton.; .
来源: N Engl J Med. 2026年394卷11期1074-1083页
Genetic deficiency of otoferlin, a protein critical to synaptic transmission by the sensory hair cells of the ear, causes congenital deafness. Medicines to treat the condition are lacking; children typically receive cochlear implants. DB-OTO is a dual adeno-associated virus 1 gene therapy that delivers human OTOF complementary DNA (encoding otoferlin) regulated by a hair cell-specific promoter.
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