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http://dx.doi.org/10.1016/j.jcin.2025.07.008 | DOI Listing |
J Pediatr Hematol Oncol
September 2025
Department of Pediatric, The University of Jordan.
Background: Rhabdomyosarcoma (RMS) typically responds well to a combination of treatments with favorable prognosis in children 1 to 9 years old. However, infants may fare worse due to receiving less aggressive local therapy for concerns about long-term effects of surgery/radiation. This study investigates the clinical characteristics, treatment approach, and survival outcomes of RMS in children under 2.
View Article and Find Full Text PDFNeurology
October 2025
Neurology, Epilepsy and Movement Disorders Unit, Bambino Gesù Children's Hospital, IRCCS, Full Member of European Reference Network on Rare and Complex Epilepsies - EpiCARE, Rome, Italy.
Objectives: Neuronal ceroid lipofuscinosis type 3 (CLN3) is a rare lysosomal storage disorder characterized by progressive neurodegeneration. No disease-modifying treatments are currently available. Miglustat, a substrate reduction therapy, has shown preclinical efficacy in CLN3 models (conference abstract).
View Article and Find Full Text PDFNeurol Neuroimmunol Neuroinflamm
November 2025
Department of Clinical Neuroscience, Karolinska Institutet, Stockholm, Sweden.
Background And Objectives: Myelitis is a relatively common clinical entity for neurologists, with diverse underlying causes. The aim of this study was to describe the incidence of myelitis, its causes, clinical presentation, and factors predicting functional outcomes and relapses.
Methods: Using the Swedish National Patient Registry, we identified all adult patients in Stockholm County between 2008 and 2018 using International Classification of Diseases, 10th Edition (ICD-10) codes likely to include myelitis.
Blood Adv
September 2025
Alfred Health and Monash University, East Melbourne, Australia.
Zanubrutinib is a next-generation covalent Bruton tyrosine kinase (BTK) inhibitor designed to provide complete and sustained BTK occupancy for efficacy across disease-relevant tissues, with fewer off-target adverse events (AEs) than other covalent BTK inhibitors. In the phase 3 ASPEN study (BGB-3111-302), comparable efficacy and a favorable safety profile versus ibrutinib were demonstrated in patients with MYD88-mutated Waldenström macroglobulinemia (WM), leading to approval of zanubrutinib for patients with WM. BGB-3111-LTE1 (LTE1) is a long-term extension study to which eligible patients, including patients from comparator treatment arms, could enroll following participation in various parent studies of zanubrutinib to treat B-cell malignancies.
View Article and Find Full Text PDFRetina
September 2025
Department of Ophthalmology, Seoul National University College of Medicine, Seoul National University Bundang Hospital, Seongnam, Korea.
Purpose: To evaluate the long-term functional and anatomical outcomes in patients with tractional lamellar macular holes who were managed without surgical intervention.
Methods: 63 eyes previously diagnosed with tractional lamellar macular hole between July 1, 2009 and January 30, 2024 without any surgical interventions were enrolled. The change in best-corrected visual acuity (BCVA), lamellar hole diameter, central retinal thickness (CRT) on Optical coherence tomography (OCT), foveal avascular zone (FAZ) areas on OCT angiography, and M-chart scores between initial and final visit were assessed.