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
Accuracy of non-invasive tests for diagnosing liver fibrosis and steatosis: A systematic review. [PDF]
Shukla SR +10 more
europepmc +1 more source
ABSTRACT Objective Neurochemical levels measured by brain MR spectroscopy (MRS) have been proposed as endpoints for clinical trials in early‐stage spinocerebellar ataxia (SCA) trials. We tested their trial‐readiness by quantifying neurochemicals in three affected brain regions in early‐stage cohorts of SCA2 and SCA3, examining their reproducibility in ...
James M. Joers +19 more
wiley +1 more source
Novel image-free arc detection method for pantograph-catenary systems based on direct DWT-ANN signal analysis. [PDF]
Elbelkasi MS +3 more
europepmc +1 more source
ABSTRACT Introduction/Objective Acute intracranial stenting during endovascular thrombectomy (EVT) for ischemic stroke requires intraprocedural antiplatelet therapy (APT) to maintain patency. However, the hemorrhagic risk of combining APT with intravenous thrombolysis (IVT) remains uncertain.
Aaron Rodriguez‐Calienes +75 more
wiley +1 more source
Passenger Lymphocyte Syndrome With Multiple Allo- and Autoantibodies Following Liver Transplantation: A Case Report. [PDF]
Marić I, Gomilšek T, Železnik K.
europepmc +1 more source
Upper Cervical Cord Area as a Biomarker of Conversion to Secondary Progressive Multiple Sclerosis
ABSTRACT Objective This study assessed whether upper cervical cord area (UCCA) measured on routine brain MRI can serve as a biomarker of conversion to SPMS. Methods This is a single‐center retrospective cohort study of RRMS patients with cross‐sectional and longitudinal analyses of clinical and MRI data. Future SPMS converters were matched by age, sex,
Nabil K. El Ayoubi +8 more
wiley +1 more source
Mechanosensitive ion channels in intestinal homeostasis and disease. [PDF]
Cao Y +8 more
europepmc +1 more source
ABSTRACT Objectives Focal cortical dysplasia (FCD) is the most common etiology of drug‐resistant epilepsy in children. Focal to bilateral tonic–clonic seizures (FBTCS) mark a high risk of drug‐resistant epilepsy and involve thalamocortical circuitry in their generation and propagation.
Hua Xie +8 more
wiley +1 more source
Recurrent Aphasic Seizures as the Initial Manifestation of LGI1 Antibody-Associated Autoimmune Limbic Encephalitis Mimicking a Transient Ischaemic Attack: A Case Report. [PDF]
Hameed A +4 more
europepmc +1 more source

