1132. AAV9-Mediated Gene Therapy for Infantile-Onset Pompe's Disease.
作者: Xiuwei Ma.;Lu Zhuang.;Wenhao Ma.;Jun Li.;Yingying Mao.;Jianhua Wang.;Xiaodong Wang.;Juan Xu.;Shuangqing Yu.;Ruijie Gu.;Yongxia Wang.;Zhongqiu Li.;Xinyang Jiang.;Sheng Zhang.;Fang He.;Xiao Yang.;Lina Zhu.;Shan Zhang.;Yanping Zhang.;Ting Li.;Qiuping Li.;Zhijie Wu.;Cengceng Zhang.;Yiying Zhang.;Xiaoyan Dong.;Hui Xiong.;Xiaobing Wu.;Zhichun Feng.
来源: N Engl J Med. 2025年392卷24期2438-2446页
Four patients with infantile-onset Pompe's disease received a single intravenous injection of an adeno-associated virus serotype 9 vector carrying codon-optimized complementary DNA encoding human acid α-glucosidase (GAA) (dose, 1.2 × 1014 vector genomes per kilogram of body weight). One patient was withdrawn from the study and subsequently died. The other patients had improvement in cardiac outcomes and motor function over a 52-week observation period. Anti-GAA antibodies were not detected in any of the patients during the observation period. Respiratory tract infections were the most common adverse events. (Funded by the National Natural Science Foundation of China and National High Level Hospital Clinical Research Funding; Chinese Clinical Trial Registry number, ChiCTR2200063229.).
1135. Once-Monthly Maridebart Cafraglutide for the Treatment of Obesity - A Phase 2 Trial.
作者: Ania M Jastreboff.;Donna H Ryan.;Harold E Bays.;Peter R Ebeling.;Mia G Mackowski.;Nisha Philipose.;Leorah Ross.;Yimeng Liu.;Cassandra E Burns.;Siddique A Abbasi.;Nicola Pannacciulli.; .
来源: N Engl J Med. 2025年393卷9期843-857页
Maridebart cafraglutide (known as MariTide) is a long-acting peptide-antibody conjugate that combines glucagon-like peptide-1 receptor agonism and glucose-dependent insulinotropic polypeptide receptor antagonism and that is intended for the treatment of obesity.
1136. Weekly Fixed-Dose Insulin Efsitora in Type 2 Diabetes without Previous Insulin Therapy.
作者: Julio Rosenstock.;Timothy Bailey.;Lisa Connery.;Eden Miller.;Cyrus Desouza.;Qianqian Wang.;Jennifer Leohr.;Alastair Knights.;Molly C Carr.;Christopher J Child.; .
来源: N Engl J Med. 2025年393卷4期325-335页
In previous treat-to-target trials, adjustments to the dose of basal insulin have been made at least weekly, according to fasting blood glucose levels. A fixed-dose regimen of insulin efsitora alfa (efsitora), a once-weekly basal insulin, may provide a benefit in adults with type 2 diabetes who have not received previous insulin therapy.
1140. Orforglipron, an Oral Small-Molecule GLP-1 Receptor Agonist, in Early Type 2 Diabetes.
作者: Julio Rosenstock.;Stanley Hsia.;Luis Nevarez Ruiz.;Sarah Eyde.;David Cox.;Wen-Shuo Wu.;Rong Liu.;Jianghao Li.;Laura Fernández Landó.;Max Denning.;Lisa Ludwig.;Yanyun Chen.; .
来源: N Engl J Med. 2025年393卷11期1065-1076页
Orforglipron is a small-molecule, nonpeptide glucagon-like peptide-1 (GLP-1) receptor agonist in clinical development for type 2 diabetes and weight management. Additional data on the efficacy and safety of orforglipron are needed.
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