Results 151 to 160 of about 285,559 (299)

Real‐World Performance of CSF Kappa Free Light Chains in the 2024 McDonald Criteria

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective Kappa free light chains (KFLCs) in the cerebrospinal fluid (CSF) have a similar performance to CSF‐restricted oligoclonal bands (OCB) for multiple sclerosis (MS) diagnosis. To help with implementation, we set out to resolve several remaining uncertainties: (1) performance in a real‐world cohort and the 2024 McDonald criteria; (2 ...
Maya M. Leibowitz, Ryan Cooper, Stefania Kaninia, Kathryn I. Challis, Will Greenway, Maria Bonello, Mahmoud Elbahnasawi, Deborah T. Shode, Dominika Gajdasik, Victor Iliev, Alison M. E. Whitelegg, Ian Galea   +11 more
wiley   +1 more source

Onasemnogene Abeparvovec in Type I Spinal Muscular Atrophy: 24‐Month Follow‐Up From the Italian Registry

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective Onasemnogene abeparvovec (OA) is an AAV9‐based gene therapy for spinal muscular atrophy type I (SMA I). Real‐world outcomes show increased response variability compared to clinical trials, and follow‐up data beyond 12–18 months are limited.
Marika Pane, Giorgia Coratti, Chiara Cutrì, Antonio Varone, Riccardo Masson, Adele D'Amico, Valeria Sansone, Sonia Messina, Federica Ricci, Chiara Ticci, Claudio Bruno, Caterina Agosto, Francesca Benedetti, Antonella Pini, Sabrina Siliquini, Massimiliano Filosto, Alberto Zambon, Ilaria Bitetti, Maria Rosaria Manna, Claudia Dosi, Riccardo Zanin, Stefano Parravicini, Roberto De Sanctis, Giulia Stanca, Michela Catteruccia, Michele Tosi, Irene Mizzoni, Emilio Albamonte, Valentina Franchino, Maria Sframeli, Ilaria Cavallina, Elena Procopio, Michele Sacchini, Simone Morando, Noemi Brolatti, Federica Trucco, Gaia Scarpini, Elena Briganti, Beatrice Berti, Concetta Palermo, Daniela Leone, Stefano C. Previtali, Eugenio Mercuri, the ITASMAC working group   +43 more
wiley   +1 more source

A Depolarizing Leak in Sodium Bicarbonate Cotransporter NBCe1 Causes Brain Edema

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objectives SLC4A4 encodes electrogenic sodium bicarbonate cotransporter NBCe1, prominently expressed in kidney and brain. Recessive loss‐of‐function variants in SLC4A4 cause proximal renal tubular acidosis, no brain edema. In the brain, NBCe1 is expressed by astrocytes, where it regulates pH and mediates astrocyte volume changes.
Quinty Bisseling, Mark D. Parker, Sven Kerst, Richard A. Pasternack, Jacob Tondreau, Marjolein Breur, Gemma M. van Rooijen‐van Leeuwen, Davide Tonduti, Ettore Salsano, Alejandra Darling, Joanna A. E. van Wijk, Susanna Törnroth‐Horsefield, Marianna Bugiani, Petra J. W. Pouwels, Quinten Waisfisz, Marjo S. van der Knaap, Rogier Min   +16 more
wiley   +1 more source

Clinical Outcomes of Patients Within the Rheumatoid Arthritis Care Pathway Cohort at a Tertiary Care Integrated Delivery Network: A Comparison to Usual Care

Arthritis Care &Research, EarlyView.
Objective We aimed to compare clinical outcomes between patients in the Allegheny Health Network rheumatoid arthritis (RA) care pathway and patients receiving usual care. Methods The care pathway initiative implements guideline‐based best practice alongside multidisciplinary team‐based care.
Tarun Sharma, Tyson S. Barrett, Teigan Dwyer, Nancy Campbell, Jessica Heintzinger, Ellen Kraemer, Adam Dore, Susan Manzi   +7 more
wiley   +1 more source

Sarilumab in Polyarticular‐Course Juvenile Idiopathic Arthritis: Dose‐Finding and One‐Year Analysis of a Phase 2b, Open‐Label, Multicenter Study

Arthritis Care &Research, Accepted Article.
Objective This study assessed sarilumab in treating patients with polyarticular‐course juvenile idiopathic arthritis (pcJIA). Methods This phase 2b, open‐label study (NCT02776735) consisted of three sequential parts (each with a core‐treatment and extension‐phase). During part 1, three doses were assessed in two weight groups (Group A/B: ≥30–60 kg/≥10–<
Fabrizio De Benedetti, Inmaculada C. Penadés, Irina Nikishina, Ivan Foeldvari, Alberto J. Spindler, Anna Kozlova, Nadina Rubio‐Pérez, Pierre Quartier, Zbigniew Żuber, Raul Barria, Daniel Clemente, Gabriel V. Cornejo, Katherine Marzan, Nancy Liu, Christine Xu, Stephen DiMartino, Angeliki Giannelou, Fei Cao‐Ghoul, Bolanle Akinlade, Lydie Baret‐Cormel   +19 more
wiley   +1 more source

vIRA Inhibition of Antiviral Necroptosis and RIPK3 Binding Are Separable Events. [PDF]

Pathogens
Ragan KB, Sridharan H, Stark AS, Ilami K, Fisher AD, Brahms ON, Kaiser WJ, Upton JW.   +7 more
europepmc   +1 more source

Pharmacokinetics, Pharmacodynamics, and Safety of Subcutaneous Belimumab in Pediatric Patients With Systemic Lupus Erythematosus: A Multicenter, Open‐Label Trial

Arthritis Care &Research, EarlyView.
Objective This study aimed to characterize the pharmacokinetics, pharmacodynamics, safety, and exploratory efficacy of subcutaneous belimumab in pediatric patients with active systemic lupus erythematosus (SLE) receiving standard therapy. Methods This single‐arm, multicenter, open‐label trial (GSK study 200908; ClinicalTrials.gov identifier ...
Hermine I. Brunner, Jordi Antón, Inmaculada Calvo‐Penadés, Richard Dimelow, Gerd Horneff, Sylvia Kamphuis, Rebecca Marino, Andre van Maurik, Kirsten Minden, Masaaki Mori, Josephine Ocran‐Appiah, Christel Wilkinson, Yuichi Yamasaki, Deepak Assudani, for the Pediatric Rheumatology Collaborative Study Group (PRCSG) and Paediatric Rheumatology International Trials Organisation (PRINTO) Network investigators   +14 more
wiley   +1 more source

Behavioural economics intervention to implement a nutrition ranking system in food pantries: Be Well cluster randomised controlled trial protocol. [PDF]

BMJ Public Health
Perez PD, Faulkner KC, Wu Y, Schwartz MB, Caspi C, Burgun R, Ortiz L, Jia J, Cheng J, Chang Y, Levy DE, Thorndike AN.   +11 more
europepmc   +1 more source

Sex Differences in Medication Discontinuation in Axial Spondyloarthritis

Arthritis Care &Research, EarlyView.
Objective We examined sex differences in medication discontinuation among patients with axial spondyloarthritis (axSpA) initiating tumor necrosis factor inhibitors (TNFi), interleukin‐17 inhibitors (IL‐17i), or JAK inhibitors (JAKi). Methods Using data from the Rheumatology Informatics System for Effectiveness (RISE) Registry (2003–2025), we assessed ...
Rachael Stovall, Cinthia Blat, Eric Roberts, Jean W. Liew, Katherine Wysham, Namrata Singh, Janna Friedly, Lianne S. Gensler, Gabriela Schmajuk, Jinoos Yazdany   +9 more
wiley   +1 more source

Genome-Edited Maize Expressing Two Native Genes Confers Broad-Spectrum Resistance to Northern Corn Leaf Blight. [PDF]

Mol Plant Pathol
Gao H, Li B, Fengler K, Yang M, Schroder M, Rahe M, Sanyour-Doyel N, Qi J, LIaca V, Beatty M, Hu WN, Barrett B, Norman B, Mo H, Leonard A, Wilson B, Meeley RB, Perugini L, Krishnamurthy N, Habben JE, Tabor G.   +20 more
europepmc   +1 more source