144. Somatic Genetic Rescue of Immunodeficiency in WHIM Syndrome.
作者: David H McDermott.;Rafael Calderon-Perez.;Zach Ormiston.;Abigail Salancy.;Bryce A Seifert.;Rachel G Moses.;Douglas B Kuhns.;Stephen M Hewitt.;David Morris.;Elena Cho.;Daniel Velez.;Priscilla E Quackenbush.;Magdalena Walkiewicz-Yvon.;Sergio M Pontejo.;Ji-Liang Gao.;Philip M Murphy.
来源: N Engl J Med. 2026年395卷1期96-98页 150. Advances in Multiple Sclerosis.
Multiple sclerosis is a chronic autoimmune disorder that affects the central nervous system, causing episodes of neurologic dysfunction and often gradual disease progression. The immune system primarily targets myelin, the protective covering of nerve fibers, leading to inflammation and damage, and secondary neurodegeneration is a major cause of long-term disability. Common symptoms include vision problems, sensory disturbances, muscle weakness, balance difficulties, and bladder dysfunction. Important advances in treatment have improved outcomes in patients with relapsing forms of multiple sclerosis, particularly through highly effective immune-modifying therapies such as CD20-targeting monoclonal antibodies. However, treatment options for progressive forms remain limited, which highlights the need for therapies that can prevent progression and promote myelin repair. Comprehensive symptom management and lifestyle support are also essential to maintaining quality of life and reducing disability.
151. Rituximab versus Ocrelizumab in Newly Diagnosed Relapsing Multiple Sclerosis.
作者: Øivind Torkildsen.;Hilde Kjelgaard Brustad.;Einar August Høgestøl.;Karl Bjørnar Alstadhaug.;Alok Bhan.;Heidi Øyen Flemmen.;Andrea Habbestad.;Rune A A Høglund.;Marissa LeBlanc.;Peter Lopen.;Åslaug Rudjord Lorentzen.;Åse Hagen Morsund.;Andreas Lossius.;Rigmor Lundby.;Gro O Nygaard.;Brit Ellen Rød.;Cecilia Smith Simonsen.;Linn Steffensen.;Hilde Torgauten.;Fredrik Piehl.;Stig Wergeland.;Kjell-Morten Myhr.; .
来源: N Engl J Med. 2026年395卷1期44-53页
Anti-CD20 monoclonal antibodies are effective for relapsing multiple sclerosis. However, data from head-to-head trials are lacking.
152. Adjuvant Pembrolizumab plus Belzutifan for Renal-Cell Carcinoma.
作者: Toni K Choueiri.;Robert J Motzer.;Jose A Karam.;Wesley Yip.;Cristina Suárez.;Dingwei Ye.;Zhisong He.;Christian Caglevic.;Tom Ferguson.;Yen-Hwa Chang.;Carlos Rojas.;Roberto Iacovelli.;Yüksel Ürün.;Elena Verzoni.;Juan Carlos Vázquez Limón.;Camillo Porta.;Robert G Uzzo.;Jae Lyun Lee.;Balaji Venugopal.;Rana R McKay.;Hans Hammers.;Hideaki Miyake.;Jad Chahoud.;Hong Liu.;Joseph E Burgents.;Manish Sharma.;Thomas B Powles.; .
来源: N Engl J Med. 2026年395卷1期32-43页
Adjuvant pembrolizumab improves disease-free and overall survival among patients with resected clear-cell renal-cell carcinoma. The hypoxia-inducible factor 2α inhibitor belzutifan has activity in advanced disease. Adjuvant pembrolizumab with belzutifan may further improve outcomes in patients with clear-cell renal-cell carcinoma at increased risk for recurrence.
156. Phase 3 Trial of Oral Infigratinib in Children with Achondroplasia.
作者: Ravi Savarirayan.;Julie Hoover-Fong.;Melita Irving.;Paul Arundel.;Josep Maria de Bergua.;Philippe M Campeau.;Toby Candler.;Benjamin T Cocanougher.;Valerie Cormier-Daire.;Thomas Edouard.;Svein O Fredwall.;Paul Harmatz.;Daniel Hoernschemeyer.;Henrik U Irgens.;Saumya Jamuar.;Peter Kannu.;Janet M Legare.;Antonio Leiva-Gea.;Helen McDevitt.;Roberta Onesimo.;John Phillips.;Mariana Del Pino.;Marie-Eve Robinson.;Massimiliano Rossi.;Mars Skae.;Leanne M Ward.;Klane K White.;Jane Schmidt.;Ted Lystig.;Ariana Salvatici.;Yun Bai.;Peter W Butler.;David van Veenhuyzen.;Daniela Rogoff.
来源: N Engl J Med. 2026年
Achondroplasia is a genetic skeletal condition caused by FGFR3 pathogenic variants. Infigratinib, an oral FGFR1-3 tyrosine kinase inhibitor, down-regulates key pathways in the pathogenesis of achondroplasia.
159. Clinical Characteristics of Patients Infected with Bundibugyo Virus, DRC 2026.
作者: Pierre Akilimali.;Dav M Ebengo.;Samuel V Scarpino.;Adrienne Amuri-Aziza.;Tony Wawina-Bokalanga.;Placide Matondo-Mbundu.;Benjamin Kanku.;Eddy Kinganda-Lusamaki.;Jean-Claude Makangara.;Daniel Mukadi-Bamuleka.;Amanda Rojek.;Moritz U G Kraemer.;Christian Ngandu.;Dieudonné Mwamba.;Placide Mbala-Kingebeni.
来源: N Engl J Med. 2026年395卷6期610-612页 |