Results 141 to 150 of about 1,250,349 (295)
ABSTRACT Background X‐linked adrenoleukodystrophy (X‐ALD) is a neurometabolic disorder caused by pathogenic variants in ABCD1, leading to slowly progressive spinal cord disease in nearly all affected men. Sensitive biomarkers to quantify disease severity and predict progression are needed for clinical care and trial design.
Eda G. Kabak +4 more
wiley +1 more source
Quantification of glycosaminoglycans in dried blood spots, and evaluation of its usefulness as a secondary newborn screening test for mucopolysaccharidoses. [PDF]
Oboshi W +27 more
europepmc +1 more source
ABSTRACT Objective To evaluate the efficacy and safety of ofatumumab in patients with myelin oligodendrocyte glycoprotein antibody–associated disease (MOGAD), and compare it with rituximab. Methods We conducted a single–center, observational study including 22 MOGAD patients treated with ofatumumab and 21 treated with rituximab.
Yuxin Fan +5 more
wiley +1 more source
Slow miniscrew-assisted palatal expansion: a clinical approach for long-term skeletal stability and enhanced nasal airway function. [PDF]
Cimen RK +5 more
europepmc +1 more source
ABSTRACT Objective This study aimed to investigate the immunological response to influenza vaccination, the incidence and severity of influenza infection, and the side effects of the vaccination in patients with ischemic stroke. Methods This prospective observational study was conducted between 2023 and 2024 at Ramathibodi Hospital.
Achiraya Pakngao +5 more
wiley +1 more source
Be AWARE: An Anti-bias Toolkit to Limit Bias in Narrative Evaluation of Medical Trainees. [PDF]
Sandgren K +3 more
europepmc +1 more source
Safety and Tolerability of Givinostat: Evidence From Real‐World and Clinical Practice
ABSTRACT Objective The aim of our study was to establish the prevalence of adverse events in a real‐world setting in boys living with Duchenne muscular dystrophy (DMD) treated with givinostat as part of an Expanded Access Program (EAP) in Italy. Methods The cohort included 90 ambulant boys, with age when treatment started between 6 and 23 years (mean ...
Marika Pane +19 more
wiley +1 more source
Clinical Outcomes of Children With Autoimmune Hepatitis in Korea: A Nationwide Multicenter Study. [PDF]
Ahn JE +16 more
europepmc +1 more source

