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Bacteriological Agents and Resistance Profiles in Neonatal Sepsis from a Poorly Regulated Antimicrobial Access Setting: Time for Action.
West Afr J Med
September 2024
Medical Microbiology & Parasitology Department, University of Ilorin, Ilorin, Nigeria. Email:
Background: Neonatal sepsis (NNS) is a known cause of morbidity and mortality especially in developing countries. The global resistance scourge may worsen the management outcomes of NNS. This study aims to determine the current profile of bacteriological agents of NNS, their resistance status and associated mortality in our setting.
View Article and Find Full Text PDFA rare case of primary testicular follicular lymphoma in a pediatric patient.
J Hematop
January 2025
Mayo Clinic, Rochester, MN, USA.
Testicular follicular lymphoma (TFL) is an exceedingly rare lymphoma that typically occurs in young male patients and is now recognized as a distinct diagnostic entity in the International Consensus Classification. TFL shows some clinicopathologic and genetic overlap with pediatric-type follicular lymphoma (PTFL). We report a case of TFL occurring in an otherwise healthy 4-year-old boy who presented with painless scrotal swelling.
View Article and Find Full Text PDFEtiology, clinical characteristics, genetic profile, and outcomes of children with refractory rickets at a referral center in India: a cohort study.
Pediatr Nephrol
January 2025
Pediatric Nephrology Services, Department of Pediatrics, Jawaharlal Institute of Postgraduate Medical Education and Research (JIPMER), Pondicherry, 605006, India.
Background: Limited research exists regarding the genetic profile, clinical characteristics, and outcomes of refractory rickets in children from India.
Methods: Patients with refractory rickets aged ≤ 18 years were enrolled. Data regarding clinical features, etiology, genotype-phenotype correlation, and estimated glomerular filtration rate (eGFR) were recorded.
High-dose glucocorticoid treatment vs. glucocorticoid replacement in immune checkpoint inhibitor associated hypophysitis (CORTICI): an open, randomised controlled trial.
Ann Med
December 2025
Division of Endocrinology and Diabetology, Department of Internal Medicine, Medical University of Graz, Graz, Austria.
Objective: One of the most severe endocrine side effects of immune checkpoint inhibitors (ICI) is hypophysitis leading to adrenal insufficiency. Recovery is rare, although it has been reported after high-dose glucocorticoid treatment. This is the first randomised study to evaluate whether hormonal recovery differs in patients treated with high-dose glucocorticoids versus glucocorticoid replacement therapy.
View Article and Find Full Text PDFPivotal trial characteristics and types of endpoints used to support Food and Drug Administration rare disease drug approvals between 2013 and 2022.
Clin Trials
January 2025
Rare Diseases Team, Office of New Drugs, Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, MD, USA.
Background/aims: Rare disease drug development faces unique challenges, such as genotypic and phenotypic heterogeneity within small patient populations and a lack of established outcome measures for conditions without previously successful drug development programs. These challenges complicate the process of selecting the appropriate trial endpoints and conducting clinical trials in rare diseases. In this descriptive study, we examined novel drug approvals for non-oncologic rare diseases by the U.
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