The New England Journal of Medicine (NEJM) RSS feed -- Search Results in Pediatrics. NEJM (https://www.nejm.org) is a weekly general medical journal that publishes new medical research findings, review articles, and editorial opinion on a wide variety of topics of importance to biomedical science and clinical practice. https://www.nejm.org/action/showPodcastsFeeds NEJM Group Sat, 13 Jun 2026 13:22:27 GMT 2026 NEJM Group en-us 1440 Observations in Down syndrome were critical to Alzheimer’s disease discoveries. But persons with Down syndrome have historically been excluded from trials relying on insights derived from their biology. https://www.nejm.org/doi/full/10.1056/NEJMp2602106?rss=searchAndBrowse https://www.nejm.org/doi/full/10.1056/NEJMp2602106 Michael S. Rafii 2026-06-13 The Convergence of Down Syndrome and Alzheimer’s Disease — Scientific and Ethical Imperatives The authors report the results of interim analyses of two phase 3 studies of exa-cel, a gene-edited cellular therapy, in children with transfusion-dependent β-thalassemia or sickle cell disease. https://www.nejm.org/doi/full/10.1056/NEJMoa2603387?rss=searchAndBrowse https://www.nejm.org/doi/full/10.1056/NEJMoa2603387 Haydar Frangoul, Josu de la Fuente, Yogi Chopra, Roland Meisel, Persis J. Amrolia, Mattia Algeri, Akshay Sharma, Maria Domenica Cappellini, Selim Corbacioglu, Antonis Kattamis, Stephan Lobitz, Mariane de Montalembert, Damiano Rondelli, Sujit Sheth, Martin H. Steinberg, Mark C. Walters, Kevin Boerner, Tina Liu, Sakellarios Zairis, William Hobbs, Stephan A. Grupp, Franco Locatelli, the CLIMB THAL-141 and CLIMB SCD-151 Study Groups* 2026-06-11 Exa-cel in Children with Transfusion-Dependent β-Thalassemia or Sickle Cell Disease In this study involving 12 patients with X-linked retinoschisis who received subretinal gene therapy, no adverse events of grade 3 or higher were reported, and changes in retinal structure were consistent with a therapeutic effect. https://www.nejm.org/doi/full/10.1056/NEJMoa2515953?rss=searchAndBrowse https://www.nejm.org/doi/full/10.1056/NEJMoa2515953 Licong Liang, Kaiqin She, Chengda Ren, Rui Li, Meng Liao, Zhiyan Tao, Fanfei Liu, Jing Su, Ming Hu, Yiliu Yang, Xiaoyue Wang, Charlotte L. Zhang, Li Bao, Qin Chen, Kang Zhang, Yuquan Wei, Yang Yang, Fang Lu 2026-06-11 Subretinal Gene Therapy for X-Linked Retinoschisis Neutralizing interleukin-10 autoantibodies were found in a subgroup of patients with inflammatory bowel disease and in no controls without IBD. Seropositivity was strongly associated with the IBD HLA-DRB1*01:03 risk allele. https://www.nejm.org/doi/full/10.1056/NEJMoa2513654?rss=searchAndBrowse https://www.nejm.org/doi/full/10.1056/NEJMoa2513654 Nima Gharahdaghi, Pai-Jui Yeh, Lourdes Ceron-Gutierrez, Helen Griffin, Hannah Gordon, Chamara Jayamanne, Alice Fracchia, Amanda Y. Chong, Alissa Walsh, Oliver Brain, Katherine Baker, Hannah Kockelbergh, Yang Luo, Martha Guevara Becerra, Katherine Vadakethala, Madeleine Coy, Ladan Kabiri, Martin Barnardo, Susanna Dunachie, Barbara Kronsteiner, Anna Adams, Darren Fowler, Qian Zhang, Laura Fachal, Carl A. Anderson, Rofaida Desoki, Marie Vibeke Vestergaard, Lone Larsen, Nicola J. Wyatt, Robert D. Lees, Robert J. Mulligan, Chaonan Dong, Mohmmed Tauseef Sharip, Siân E. Faustini, Adrian Shields, Julia Pakpoor, Bushra Naz, Alex Richter, Jack Satsangi, Christopher A. Lamb, Miles Parkes, Fiona Powrie, Alexander J. Mentzer, Aleksejs Sazonovs, Tine Jess, Paul Klenerman, Simon Travis, Sophie Hambleton, Rainer Doffinger, Holm H. Uhlig 2026-06-11 Interleukin-10 Autoantibodies and HLA-DRB1*01:03 in Inflammatory Bowel Disease An 11-year-old girl presented with a 1-month history of right thigh pain that worsened at night. Radiographs of the knee revealed a metaphyseal osteoblastic lesion with a sunburst appearance. https://www.nejm.org/doi/full/10.1056/NEJMicm2600191?rss=searchAndBrowse https://www.nejm.org/doi/full/10.1056/NEJMicm2600191 Hsueh-Min Kuo, Wei-Ming Chen 2026-06-11 Osteosarcoma A 14-year-old girl was admitted to the hospital because of hypertension and headache. She had hypokalemia, an elevated blood level of C-reactive protein, and an elevated erythrocyte sedimentation rate. A diagnosis was made. https://www.nejm.org/doi/full/10.1056/NEJMcpc2517866?rss=searchAndBrowse https://www.nejm.org/doi/full/10.1056/NEJMcpc2517866 Rachel R. Osborn, Jonathan S. Hausmann, Weizhen Tan, Evan J. Zucker 2026-06-11 Case 16-2026: A 14-Year-Old Girl with Hypertension Having inherited a strong sense of professionalism from her mother and female physician mentors, a trainee finds the realities of juggling motherhood and medicine more difficult than she expected. https://www.nejm.org/doi/full/10.1056/NEJMp2513897?rss=searchAndBrowse https://www.nejm.org/doi/full/10.1056/NEJMp2513897 Sarah Calvert 2026-06-11 Carrier The authors describe the scientific foundations of a study implicating autoantibodies against interleukin-10 in persons with inflammatory bowel disease. https://www.nejm.org/doi/full/10.1056/NEJMe2603635?rss=searchAndBrowse https://www.nejm.org/doi/full/10.1056/NEJMe2603635 Paul Bastard, Jean-Laurent Casanova 2026-06-11 Science behind the Study: Autoantibodies against Cytokines — From Infection to Inflammation If the current cholera pandemic, which began in 1961, is ever to end, the world will need better cholera vaccines that are effective in young children and global access to safe water and adequate sanitation. https://www.nejm.org/doi/full/10.1056/NEJMp2600873?rss=searchAndBrowse https://www.nejm.org/doi/full/10.1056/NEJMp2600873 Edward T. Ryan, Firdausi Qadri, Julia A. Lynch 2026-06-11 Global Cholera-Control Efforts — Progress and Remaining Challenges A neuroepithelial tumor occurred in a child who had previously received AAV gene therapy for mucopolysaccharidosis type I. Analysis of tumor tissue showed the integration of rearranged AAV vector elements in the gene PLAG1. https://www.nejm.org/doi/full/10.1056/NEJMoa2601608?rss=searchAndBrowse https://www.nejm.org/doi/full/10.1056/NEJMoa2601608 Rebecca C. Ahrens-Nicklas, Chelsea Kotch, Aoife M. Roche, John K. Everett, Shantan Reddy, Mariarita Santi, Peter J. Madsen, Cristina Martos-Rus, Carolyn M. Yrigollen, Juliana C. Small, Sarah McCague, Beverly L. Davidson, Benjamin J. Samelson-Jones, Lea F. Surrey, Marilyn Li, Can Ficicioglu, Raymond Wang, Frederic D. Bushman, Lindsey A. George 2026-06-04 Brief Report: Neuroepithelial Tumor with AAV Integration after Intracisternal Magna Vector Delivery Vaccine hesitancy is often driven by safety concerns. Clinician recommendations, presumptive communication, and empathy improve uptake; maintaining trust supports future acceptance and community protection. https://www.nejm.org/doi/full/10.1056/NEJMcp2516616?rss=searchAndBrowse https://www.nejm.org/doi/full/10.1056/NEJMcp2516616 Sean T. O’Leary, Margie Danchin 2026-06-04 Childhood Vaccine Hesitancy The prevalence of wasting among children in Gaza tracks with Israeli blockades on aid, but war may lead to famine even without such policies. How should starvation be prevented and treated in wartime? https://www.nejm.org/doi/full/10.1056/NEJMp2602465?rss=searchAndBrowse https://www.nejm.org/doi/full/10.1056/NEJMp2602465 Kevin Stephenson, Christa Lolley, Mark Manary 2026-06-04 War and Famine A 44-year-old man presented with a 4-year history of choking episodes while eating. Endoscopy revealed narrowing of the upper esophagus by a pulsating, cordlike structure in the posterior wall (shown in a video). https://www.nejm.org/doi/full/10.1056/NEJMicm2600585?rss=searchAndBrowse https://www.nejm.org/doi/full/10.1056/NEJMicm2600585 Ishita Dubey, Neha Wadhwa 2026-06-04 Dysphagia Lusoria In an African study of severe malaria in children with partially artemisinin-resistant parasites with PfK13 mutations, artesunate did not lead to worse outcomes, which suggests that parenteral artesunate may still be used. https://www.nejm.org/doi/full/10.1056/NEJMc2517274?rss=searchAndBrowse https://www.nejm.org/doi/full/10.1056/NEJMc2517274 Kathryn Maitland, Maurice Okao, Melissa D. Conrad, Tonny Etwop, Modester Akite, Emmanuel Oguda, Florence Alaroker, Denis Aromut, Rita Muhindo, Sophie Uyoga, Peter Olupot-Olupot, Christabel Mogaka, Roisin Connon, Thomas Katairo, Martin Okitwi, Jessica Briggs, Diana M. Gibb, Nicholas P.J. Day, Nicholas J. White, Thomas N. Williams, Arjen M. Dondorp, A. Sarah Walker, Elizabeth C. George, Philip J. Rosenthal, for the SMAART-CHARISMA group† 2026-06-03 Intravenous Artesunate in Artemisinin-Resistant Severe Malaria in Uganda In a trial involving children with Chiari type I malformation and syringomyelia, the percentage with surgical complications did not differ significantly between posterior fossa decompression with duraplasty and decompression alone. https://www.nejm.org/doi/full/10.1056/NEJMoa2402821?rss=searchAndBrowse https://www.nejm.org/doi/full/10.1056/NEJMoa2402821 David D. Limbrick Jr., Chevis N. Shannon, Emine O. Bayman, Thanda Meehan, Marisa Kallem, Laurie L. Ackerman, P. David Adelson, Raheel Ahmed, Gregory Albert, Philipp R. Aldana, Tord D. Alden, Richard C.E. Anderson, Lissa C. Baird, David Bauer, Tammy Bethel-Anderson, Karin Bierbrauer, Douglas L. Brockmeyer, Joshua J. Chern, Daniel E. Couture, David J. Daniels, Robert C. Dauser, Brian J. Dlouhy, Susan R. Durham, Richard G. Ellenbogen, Ramin Eskandari, Stephen R. Gannon, Gerald A. Grant, Patrick C. Graupman, Jacob K. Greenberg, Stephanie Greene, Jeffrey P. Greenfield, Naina L. Gross, Daniel J. Guillaume, Todd C. Hankinson, Gregory Heuer, Mark Iantosca, Bermans J. Iskandar, Eric M. Jackson, George I. Jallo, James M. Johnston, Robert F. Keating, Michael P. Kelly, Elaine Kennedy, Mark D. Krieger, Abhaya V. Kulkarni, Rick Labuda, Jeffrey R. Leonard, Cormac O. Maher, Francesco T. Mangano, J. Gordon McComb, Sean D. McEvoy, Robert C. McKinstry, Arnold H. Menezes, Toba N. Niazi, Jerry Oakes, Greg Olavarria, Brent R. O’Neill, Dorothy Poppe, John Ragheb, Samuel Reeves, Lisa Reynolds, Nathan R. Selden, Manish N. Shah, Joshua S. Shimony, Matthew D. Smyth, Sandy Spears, Scellig S.D. Stone, Jennifer M. Strahle, Mandeep Tamber, Gina Tramelli, Gerald F. Tuite, Elizabeth C. Tyler-Kabara, Haley Vance, Scott D. Wait, John C. Wellons III, William E. Whitehead, Alexander T. Yahanda, Yan Yan, Ralph G. Dacey Jr., Tae Sung Park, James C. Torner, the Park-Reeves Syringomyelia Research Consortium Investigators 2026-05-28 Decompression with or without Duraplasty for Chiari I and Syringomyelia The new Dietary Guidelines for Americans represent progress in some areas but raise concerns about transparency, internal consistency, practical implementation, and ignoring of key scientific evidence. https://www.nejm.org/doi/full/10.1056/NEJMp2600579?rss=searchAndBrowse https://www.nejm.org/doi/full/10.1056/NEJMp2600579 Deirdre K. Tobias, Frank B. Hu 2026-05-28 The 2025–2030 Dietary Guidelines for Americans — Progress, Pitfalls, and the Path Forward After a physician pronounces his 15-year-old patient dead over the phone, he respects her parents’ silence, though it leaves him isolated in his grief and craving some form of closure. https://www.nejm.org/doi/full/10.1056/NEJMp2513303?rss=searchAndBrowse https://www.nejm.org/doi/full/10.1056/NEJMp2513303 James A. Feinstein 2026-05-28 When Closure Finds You Ugandan children with sickle cell anemia who received 10 years of hydroxyurea therapy had sustained laboratory and clinical benefits, improved growth, and few toxic effects. https://www.nejm.org/doi/full/10.1056/NEJMc2603063?rss=searchAndBrowse https://www.nejm.org/doi/full/10.1056/NEJMc2603063 Russell E. Ware, Robert O. Opoka, Teresa Latham, Phillip Kasirye, Priscilla Kasembo, Heather Hume, Isaac Birungi, Norah Kadde, Bill Wambaka, Maria Nakafeero, Andrea L. Conroy, George Tomlinson, Adam Lane, Chandy John 2026-05-28 Ten Years of Hydroxyurea for Ugandan Children with Sickle Cell Anemia Azithromycin did not lead to a greater reduction in symptom severity than placebo among preschool children presenting to the emergency department with wheezing, regardless of whether they tested positive for pathogenic bacteria. https://www.nejm.org/doi/full/10.1056/NEJMoa2516505?rss=searchAndBrowse https://www.nejm.org/doi/full/10.1056/NEJMoa2516505 Kurt R. Denninghoff, T. Charles Casper, Joseph J. Zorc, Richard M. Ruddy, Sarah Satola, Wendi-Jo Wendt, Claudia R. Morris, Melissa M. Tavarez, Matthew J. Lipshaw, Maria Y. Kwok, Jo-Ann O. Nesiama, Kyle A. Nelson, Michael Webb, Fernando D. Martinez, the PECARN AZ-SWED Trial Study Group* 2026-05-18 Azithromycin for Preschoolers with Wheezing in the Emergency Department An analysis of current causes of deaths among U.S. children and adolescents underscores the need for sustained investment in prevention, research on injury and overdose treatments, and readiness in emergency settings. https://www.nejm.org/doi/full/10.1056/NEJMc2600445?rss=searchAndBrowse https://www.nejm.org/doi/full/10.1056/NEJMc2600445 Caroline Raymond-King, Molly M. Jeffery, Edward R. Melnick 2026-05-14 Current Causes of Death among Children and Adolescents in the United States