Results 41 to 50 of about 98,771 (295)
Estimation of conditional average treatment effects on distributed confidential data
45 pages, 12 ...
Yuji Kawamata +4 more
openaire +3 more sources
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
Undersmoothing Causal Estimators With Generative Trees
Average causal effects are averages of (heterogeneous) individual treatment effects (ITEs) taken over the entire target population. The estimation of average causal effects has been studied in depth, but averages are insufficient for more individualised ...
Damian Machlanski +2 more
doaj +1 more source
Evaluation of Educational Interventions Based on Average Treatment Effect: A Case Study
Relative to randomized controlled trials (RCTs) with privacy and ethical concerns, observational studies are becoming dominant in education research. In an observational study, it is necessary and important to correctly evaluate the effects of different ...
Jingyu Liang, Jie Liu
doaj +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 +9 more
wiley +1 more source
ALDOA Promotes Glycolysis and NLRP3/GSDMD Pyroptosis to Accelerate ALS Progression
ABSTRACT Objective Amyotrophic lateral sclerosis (ALS) is characterized by progressive motor neuron degeneration. Glycolytic dysregulation is implicated in disease progression, yet the underlying mechanisms remain unclear. This study investigates how Aldolase A (ALDOA) drives ALS progression through glycolysis‐mediated motor neuron pyroptosis.
Kaixin Yan +9 more
wiley +1 more source
Nonparametric estimation of conditional incremental effects
Conditional effect estimation has great scientific and policy importance because interventions may impact subjects differently depending on their characteristics.
McClean Alec +2 more
doaj +1 more source
Choice of non-inferiority (NI) margins does not protect against degradation of treatment effects on an average--an observational study of registered and published NI trials. [PDF]
OBJECTIVE:NI margins have to be chosen appropriately to control the risk of degradation of treatment effects in non-inferiority (NI) trials. We aimed to study whether the current choice of NI margins protects sufficiently against a degradation of ...
Beryl Primrose Gladstone, Werner Vach
doaj +1 more source
There is empirical evidence of an association between periodontitis and coronary heart disease (CHD). However, it is uncertain whether periodontal treatment in CHD patients might lead to reduced healthcare costs. This study aims to assess the association
Katja Blaschke +4 more
doaj +1 more source

