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 Wed, 09 Sep 2026 00:58:53 GMT 2026 NEJM Group en-us 1440 During Operation Metro Surge in Minneapolis, volunteer health care professionals quickly improvised a new system, often without institutional authorization, to continue to care for terrified patients. https://www.nejm.org/doi/full/10.1056/NEJMp2607981?rss=searchAndBrowse https://www.nejm.org/doi/full/10.1056/NEJMp2607981 Munira Maalimisaq, Jennifer Needle-Suarez 2026-09-05 When Fear Became the Barrier to Care — Lessons of a Clinical Rapid Response during Operation Metro Surge In children with septic shock, no significant difference was seen in the incidence of death, new renal-replacement therapy, or persistent kidney dysfunction with balanced fluid as compared with 0.9% saline for fluid resuscitation. https://www.nejm.org/doi/full/10.1056/NEJMoa2601969?rss=searchAndBrowse https://www.nejm.org/doi/full/10.1056/NEJMoa2601969 Fran Balamuth, Scott L. Weiss, Elliot Long, Graham C. Thompson, Amanda S. Artis, Atzael B. Campos, Meredith L. Borland, Stuart R. Dalziel, Adriana Yock-Corrales, Ruchi Singh, Amanda Williams, Beata Mickiewicz, Christopher P. Hickey, Julie C. Fitzgerald, Benjamin L. Laskin, Robert W. Hickey, Michelle Eckerle, Waleed Alqurashi, Elizabeth Alpern, Lilliam Ambroggio, Mohamed Badawy, Shannon Baumer-Mouradian, Simon Berthelot, Lindsay D. Clukies, Simon Craig, Sarah J. Curtis, Adrienne L. Davis, Susan Duffy, Matthew A. Eisenberg, Jason G. Emsley, Ara Festekjian, Shane George, Rebecca Green, Karen E. Gripp, Priya G. Jain, Shefali Jani, Gary I. Joubert, Pavan Judge, April Kam, Amit Kochar, Ioannis Koutroulis, Maria Y. Kwok, Roni D. Lane, Anna Lithgow, Julia Lloyd, Karim Mansour, Julie K. McManemy, Claudia Morris, Natalie Phillips, Arjun Rao, Alexander Rogers, Anupam Sehgal, Yasaman Shayan, Jonathan Silverman, Eunicia Tan, Neil G. Uspal, Cheryl Vance, Emma Whyte, Jing Huang, Stephen B. Freedman, Franz E. Babl, Nathan Kuppermann, the PRoMPT BOLUS Investigators of the PECARN, PERC, and PREDICT Networks* 2026-09-03 Balanced Fluid or 0.9% Saline in Children Treated for Septic Shock In this phase 3, randomized trial involving children with achondroplasia (3 to 17 years of age), once-daily oral infigratinib for 1 year significantly increased the annualized height velocity as compared with placebo. https://www.nejm.org/doi/full/10.1056/NEJMoa2604565?rss=searchAndBrowse https://www.nejm.org/doi/full/10.1056/NEJMoa2604565 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 III, 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 2026-09-03 Phase 3 Trial of Oral Infigratinib in Children with Achondroplasia Achondroplasia, the most common skeletal dysplasia, is characterized by impaired longitudinal growth resulting in disproportionate short stature as well as craniofacial abnormalities, spinal stenosis, and increased mortality during infancy from cervicomedullary compression. This condition is caused by mutations in the gene encoding fibroblast growth factor receptor 3 (FGFR3) ,... https://www.nejm.org/doi/full/10.1056/NEJMe2609749?rss=searchAndBrowse https://www.nejm.org/doi/full/10.1056/NEJMe2609749 Isidro B. Salusky, Harald Jüppner 2026-09-03 Infigratinib in Achondroplasia — A Potentially Giant Step Forward Improving the clinical treatment of patients with sepsis remains a major focus of critical care physicians worldwide.1,2 To date, few studies in either the pediatric or adult literature have established universal management principles that reliably improve outcomes, which attests to the heterogeneity of this syndrome. Fluid resuscitation is... https://www.nejm.org/doi/full/10.1056/NEJMe2609856?rss=searchAndBrowse https://www.nejm.org/doi/full/10.1056/NEJMe2609856 Michael F. O’Connor, Juliane Bubeck-Wardenburg 2026-09-03 Fluid Resuscitation in Patients with Sepsis — For Whom the Balance Tolls Azithromycin did not lead to a greater reduction in symptom severity than placebo among preschool children presenting to the ED 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* 2026-08-20 Azithromycin for Preschoolers with Wheezing in the Emergency Department Acute wheezing attacks are common in preschool-age children. These attacks lead to impaired quality of life1 and are associated with trajectories of poor lung function that may be lifelong, especially in children who are exposed to tobacco smoke and have allergic sensitization.2 Despite increased focus on... https://www.nejm.org/doi/full/10.1056/NEJMe2606675?rss=searchAndBrowse https://www.nejm.org/doi/full/10.1056/NEJMe2606675 Andrew Bush, Sejal Saglani 2026-08-20 No to Azithromycin for Preschoolers with Acute Wheezing in the Emergency Department This article describes gene editing in a mouse model of a peroxisomal disorder, as well as the broader landscape, including regulation, of the treatment of rare genetic disease through gene editing. https://www.nejm.org/doi/full/10.1056/NEJMcibr2606991?rss=searchAndBrowse https://www.nejm.org/doi/full/10.1056/NEJMcibr2606991 Rebecca C. Ahrens-Nicklas 2026-08-13 Gene Editing, Peroxisomal Disorders, and Treatments for Rare Disease A study noted a sharp increase in leucovorin prescriptions dispensed to children (≤17 years of age) after a plan was announced to update the FDA drug label to include cerebral folate deficiency, which may include autism symptoms. https://www.nejm.org/doi/full/10.1056/NEJMc2602260?rss=searchAndBrowse https://www.nejm.org/doi/full/10.1056/NEJMc2602260 Kao-Ping Chua, Sijia He, Rena M. Conti 2026-08-13 Leucovorin Dispensing to U.S. Children in 2025 In a child with advanced nephroblastoma, T-cell therapy targeting preferentially expressed antigen in melanoma led to a 69% tumor reduction, with molecular, metabolic, and histologic evidence of complete remission at 200 days. https://www.nejm.org/doi/full/10.1056/NEJMc2605154?rss=searchAndBrowse https://www.nejm.org/doi/full/10.1056/NEJMc2605154 Katharina Mair, Corinne Rossi, Jens H. Westhoff, Thomas G.P. Grünewald, Jens-Peter Schenk, Alexandra Tuch, Esther Wahlbrink, Stefanie Volz, Kendra K. Maass, Sophia Scheuermann, Simon Krost, Fabienne Engelmann, Barbara C. Jones, Joachim B. Kunz, Inga Harting, Erik Winter, Patrick Guenther, Anita Schmitt, Carsten Müller-Tidow, Liane Preussner, Cedrik M. Britten, Robert J. Autry, David T.W. Jones, Stefan M. Pfister, Dirk Jäger, Andreas E. Kulozik, Olaf Witt, Patrick Schmidt, Christian M. Seitz 2026-08-13 PRAME-Specific T-Cell Therapy in Advanced Pediatric Nephroblastoma In a patient with Duchenne muscular dystrophy, cardiotoxic effects developed after treatment with delandistrogene moxeparvovec. Microdystrophin levels in the heart and skeletal muscle were less than 1% of normal dystrophin levels. https://www.nejm.org/doi/full/10.1056/NEJMc2518477?rss=searchAndBrowse https://www.nejm.org/doi/full/10.1056/NEJMc2518477 Benjamin J. Samelson-Jones, Cristina Martos-Rus, Carolyn M. Yrigollen, Luis Tecedor, Juliana C. Small, Susan E. Matesanz, Connor T. Watson, Andrew R. Vanden Heuvel, Sean Carrig, Sabrina W. Yum, John F. Brandsema, Kimberly Lin, Carol Wittlieb-Weber, Angela N. Viaene, Pierre P. Russo, Sara Partington, Katie Huynh, Tiffany Seneviratne, Silvia Juarez Rojas, Bhavya S. Doshi, Perry Shieh, Kevin M. Flanigan, Emma Frair, Stefan Nicolau, Michael W. Lawlor, Julie M. Crudele, Beverly L. Davidson, Lindsey A. George 2026-08-06 Cardiotoxic Effects and Microdystrophin Expression after Gene Therapy for DMD Alternative complement pathway overactivation contributes to IgA nephropathy. In a placebo-controlled trial, iptacopan, a complement factor B inhibitor, significantly slowed kidney-function decline in patients with IgA nephropathy. https://www.nejm.org/doi/full/10.1056/NEJMoa2600743?rss=searchAndBrowse https://www.nejm.org/doi/full/10.1056/NEJMoa2600743 Jonathan Barratt, Necmi Eren, Naoki Kashihara, Bart Maes, Dana V. Rizk, Brad Rovin, Hernán Trimarchi, Hong Zhang, Weiming Wang, Ismail Kocyigit, Chuanming Hao, Vladimir Tesař, Kenan Turgutalp, Li Yang, Guangqun Xing, Valter Duro Garcia, Seung Hyeok Han, Wanhong Lu, Antonio Pisani, Julia Weinmann-Menke, Frank Eitner, Nicolas Guerard, Dmytro Butylin, Luca Monaco, Emil Scosyrev, Annabel Magirr, Ronny Renfurm, Thomas Hach, Vlado Perkovic, the APPLAUSE-IgAN Study Group* 2026-07-30 Iptacopan in IgA Nephropathy — Final 24-Month Data The Trump administration’s legal theories about gender-affirming care, if accepted, could shift the equilibrium of medical decision making in the United States in important ways. https://www.nejm.org/doi/full/10.1056/NEJMp2605975?rss=searchAndBrowse https://www.nejm.org/doi/full/10.1056/NEJMp2605975 Benjamin A. Barsky, Brendan Reid, Christopher Robertson 2026-07-30 Medical Standards by Federal Fiat This study supports enzymatic cross-correction of neurons and oligodendrocytes by atidarsagene autotemcel, a gene therapy that prevents or slows neurodegeneration in children with early-onset metachromatic leukodystrophy. https://www.nejm.org/doi/full/10.1056/NEJMc2505002?rss=searchAndBrowse https://www.nejm.org/doi/full/10.1056/NEJMc2505002 Vasco Meneghini, Valeria Calbi, Filippo Casalini, Elisabeth Mangiameli, Francesco Morena, Marco Piccoli, Ilaria Laface, Ilaria Rossomanno, Francesca Ornaghi, Andrea Ghiroldi, Paola M.V. Rancoita, Francesca Fumagalli, Sabata Martino, Luigi Anastasia, Luigi Naldini, Alessandro Aiuti, Angela Gritti 2026-07-30 Cross-Correction in HSC Gene Therapy for Metachromatic Leukodystrophy In this trial, among adolescents with obstructive hypertrophic cardiomyopathy, the cardiac myosin inhibitor mavacamten led to a significantly greater reduction in left ventricular outflow tract obstruction than placebo. https://www.nejm.org/doi/full/10.1056/NEJMoa2601103?rss=searchAndBrowse https://www.nejm.org/doi/full/10.1056/NEJMoa2601103 Joseph W. Rossano, Charles Canter, Cordula M. Wolf, Andrew Papez, Marta Gambra, Randall M. Bryant, Juan Alejos, Michael McCulloch, Georgia Sarquella Brugada, Matthew J. Bock, Aamir Jeewa, F. Bennett Pearce, Milind Y. Desai, Nicholas Favatella, Atefeh Javidialsaadi, Van Phung, Thomas Rano, Lin Zhu, Joshua L. Dyme, Seema Mital, the SCOUT-HCM Investigators* 2026-07-23 Mavacamten in Adolescents with Obstructive Hypertrophic Cardiomyopathy Fibromyalgia is associated with widespread pain, sleep difficulty, fatigue, and cognitive symptoms. Treatment includes education and nonpharmacologic interventions; selected drugs may help, but opioid agents are not recommended. https://www.nejm.org/doi/full/10.1056/NEJMcp2411656?rss=searchAndBrowse https://www.nejm.org/doi/full/10.1056/NEJMcp2411656 David A. Williams, Daniel J. Clauw 2026-07-16 Fibromyalgia Medication management in autism requires clinicians to navigate uncertainty while respecting the experiences of autistic people and their families. Shared decision making can be a helpful approach. https://www.nejm.org/doi/full/10.1056/NEJMp2602649?rss=searchAndBrowse https://www.nejm.org/doi/full/10.1056/NEJMp2602649 Yaara Zisman-Ilani, Ron Walfisch, Jeremy Veenstra-VanderWeele, David S. Mandell 2026-07-16 Pharmacotherapeutic Decisions in Autism A 6-month-old baby boy was transferred to a quaternary children’s hospital for seizures. A radiograph of a hand and wrist showed dense, transverse bands of metaphyseal sclerosis in the radius, ulna, metacarpals, and phalanges. https://www.nejm.org/doi/full/10.1056/NEJMicm2603074?rss=searchAndBrowse https://www.nejm.org/doi/full/10.1056/NEJMicm2603074 Jason Haines, Tony Huynh 2026-07-16 Lead Lines in Severe Lead Poisoning Among participants 4 to 66 years of age with acquired hypothalamic obesity, setmelanotide, a melanocortin-4 receptor agonist, led to significantly greater reductions in body-mass index and hunger than placebo at 52 weeks. https://www.nejm.org/doi/full/10.1056/NEJMoa2512275?rss=searchAndBrowse https://www.nejm.org/doi/full/10.1056/NEJMoa2512275 Jennifer L. Miller, Hanneke M. van Santen, Susan A. Phillips, Jill Hamilton, Jens Aberle, Thozhukat Sathyapalan, Zainaba Mohamed, Shana E. McCormack, Ashley H. Shoemaker, Megan M. Kelsey, Luma Ghalib, Guenter Stalla, Vidhu V. Thaker, Reema Habiby, Katie Larson Ode, Martin Wabitsch, Mehul Dattani, M. Jennifer Abuzzahab, Hussein Abdullatif, Ryan Morgan, Margaret Stefater-Richards, Vanita R. Aroda, Carsten Friedrich, Joan C. Han, Hiraku Ono, Keisuke Nagasaki, Tomohiro Tanaka, Tsuyoshi Isojima, Hiroshi Arima, Cecilia Scimia, Guojun Yuan, Hermann L. Müller, Christian L. Roth, the TRANSCEND Trial Group* 2026-07-09 Setmelanotide for the Treatment of Acquired Hypothalamic Obesity The author describes the scientific basis for a trial of an MC4R agonist in persons with acquired hypothalamic obesity. Those who received the drug had reductions in body-mass index and hunger. https://www.nejm.org/doi/full/10.1056/NEJMe2606217?rss=searchAndBrowse https://www.nejm.org/doi/full/10.1056/NEJMe2606217 I. Sadaf Farooqi 2026-07-09 Science behind the Study: Treating Acquired Hypothalamic Obesity