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 +43 more
wiley +1 more source
Protocol for a pragmatic trial comparing navigator and automated virtual interventions for guaranteeing adherence to fecal immunochemical testing (NAVIGATE-FIT). [PDF]
Jansen E +7 more
europepmc +1 more source
Quantifying the Impact of Ocrelizumab on Paramagnetic Rim Lesions in Multiple Sclerosis
ABSTRACT Paramagnetic rim lesions (PRLs) are a subset of chronic active multiple sclerosis (MS) lesions marked by iron‐laden microglia and macrophages. Ocrelizumab, a monoclonal antibody targeting CD20+ B cells, suppresses acute MS activity, but its effect on PRLs remains unclear. In a longitudinal study of 29 ocrelizumab‐treated patients with at least
Kimberly H. Markowitz +8 more
wiley +1 more source
Digital Cognitive Behavioral Therapy for Insomnia in Cancer Pain Patients: Protocol for a Prospective, Randomized Controlled Trial. [PDF]
Lan X +5 more
europepmc +1 more source
ABSTRACT Objective This analysis evaluates the effect of successful reperfusion on functional outcomes after MT, stratified by admission National Institutes of Health Stroke Scale (NIHSS) and Alberta Stroke Program Early CT Score (ASPECTS) as surrogates for clinical‐core mismatch, using multicenter registry data.
Felix Schlicht +53 more
wiley +1 more source
Analysis of clinical research coordinator (CRC) deviation from assigned responsibilities in Sichuan, China based on empirical investigation. [PDF]
Shi S, Qin S, Yu Q.
europepmc +1 more source
Clinical Validation of Plasma p‐217tau in Neurological Diseases
ABSTRACT Objective Plasma p‐217tau is a minimally invasive but specific biomarker for diagnosing Alzheimer's disease (AD). However, its disease specificity remains to be clinically evaluated. We validated the reliability of the p‐217tau biomarker in 12 other neurological diseases.
Takeshi Kawarabayashi +13 more
wiley +1 more source
Targeting Investigation and Treatment in Type 2 Myocardial Infarction: A Pilot Randomized Controlled Trial. [PDF]
Taggart C +22 more
europepmc +1 more source
Study protocol for a randomised controlled trial of dance-movement therapy for adolescents with depression. [PDF]
Valjakka R +5 more
europepmc +1 more source

