Results 51 to 60 of about 90,086 (258)
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
CSF Monoamine Metabolites and Cognitive Trajectory in Early Parkinson's Disease
ABSTRACT Background Imaging and postmortem studies indicate that abnormalities in monoaminergic neurotransmission contribute to cognitive impairment in Parkinson's disease (PD). However, it remains uncertain if cerebrospinal fluid (CSF) monoamine metabolites can serve as biomarkers of cognitive decline in early PD.
Jing‐Yu Shao +7 more
wiley +1 more source
Background: The Zarit Burden Interview (ZBI) is a validated tool for assessing caregiver burden, while the Distress Thermometer (DT) measures patient distress.
Letizia Franzini +5 more
doaj +1 more source
Problematic Internet Use in Frontotemporal Dementia: A Case Series
ABSTRACT The present study investigated problematic internet use (PIU) among 61 patients with frontotemporal dementia (FTD) compared to a cohort of 354 patients with mild cognitive impairment (MCI) and Alzheimer's dementia. PIU was identified in 22.9% of FTD patients compared to only 0.8% of AD patients (p < 0.001). Behaviors included compulsive social
Daniele Urso +9 more
wiley +1 more source
Abstrak. Skizofrenia merupakan gangguan mental berat yang ditandai dengan gangguan yang mendalam pada proses berfikir, berbahasa, persepsi, dan pengalaman psikotik berupa mendengar suara-suara yang tidak lazim ataupun delusi.
Dianita Permatasari +2 more
doaj +1 more source
ABSTRACT Objective Digital technologies hold promise for transforming healthcare by enhancing personalized treatments and offer valuable opportunities to improve patient care. Here, we evaluated several novel, self‐administered, home‐based, digital endpoints for their association with corresponding conventional standard clinical measures (primary) in ...
Arne Mueller +14 more
wiley +1 more source
Background Socioeconomic position (SEP) is an important contextual factor in the Stress Process Model of caregiving. However, the basic assumption that low SEP is associated with greater caregiver burden has so far lacked empirical support. The objective
Hannah Tough +4 more
doaj +1 more source
ABSTRACT Objective Variants in SLC6A1, encoding the GABA transporter 1 (GAT‐1), cause epilepsy, autism spectrum disorder, and developmental delay via loss of GABA uptake, impaired trafficking, and ER retention. We previously found that 4‐Phenylbutyrate (PBA), an FDA‐approved drug, restores GABA uptake and reduces seizures in SLC6A1‐related disorders ...
Melissa B. DeLeeuw +5 more
wiley +1 more source
Onasemnogene Abeparvovec in Patients With SMA: Interim Results of the RESTORE Registry in Japan
ABSTRACT Objective There are limited real‐world data regarding the safety and effectiveness of onasemnogene abeparvovec (OA; Zolgensma) infusion, a one‐time gene replacement therapy, for Japanese patients with spinal muscular atrophy (SMA). We aimed to improve understanding of the real‐world outcomes for OA in Japan.
Kayoko Saito +8 more
wiley +1 more source
Frontal Function, Disability and Caregiver Burden in Elderly Patients With Major Depressive Disorder
Caregivers of patients with late-life major depressive disorder experience a significant level of general caregiver burden. Disability in patients is possibly one of the origins of caregiver burden.
Hui-Mei Chen +7 more
doaj +1 more source

