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
Lactate, mean platelet volume, and nSOFA as combined predictors of perforated necrotizing enterocolitis and mortality in preterm infants: a retrospective cohort study. [PDF]
Huang D +9 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
Persistence of proinsulin-only secretion in people with long-duration diabetes and minimal residual insulin secretion. [PDF]
Metghalchi S +11 more
europepmc +1 more source
The Case of a 55‐Year‐Old Man With Leg Weakness
Annals of Clinical and Translational Neurology, EarlyView.
Ada Breitenbucher +3 more
wiley +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
Residual inflammatory risk post STEMI: high prevalence despite LDL-C control and association with other secondary prevention targets. [PDF]
Hassan A +9 more
europepmc +1 more source
Multiple Sclerosis Relapse Activity After Ozanimod Discontinuation in DAYBREAK Trial Participants
Multiple Sclerosis Relapse Activity After Ozanimod Discontinuation in DAYBREAK Trial Participants. ABSTRACT Objective Return of disease activity is expected when patients discontinue disease‐modifying therapy (DMT) for multiple sclerosis (MS). Some MS DMTs are associated with higher‐than‐expected disease activity (rebound) after discontinuation.
Ralf Gold +12 more
wiley +1 more source
Global burden trends and menopause timing associations for gynecological and breast cancers in postmenopausal women. [PDF]
Li S +5 more
europepmc +1 more source

