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
Development and Preliminary Validation of the Chinese Medicine Interventional Clinical Trials Research Question Importance Tool (CMICT-RQIT). [PDF]
Cheng T, Yu D, Qiu X, Li G, Wen Z.
europepmc +1 more source
A Two‐Stage Questionnaire and Actigraphy Screening for iRBD in a Multicenter Retrospective Cohort
ABSTRACT Objective Isolated rapid‐eye‐movement sleep behavior disorder is a prodromal marker of synucleinopathies. However, most cases remain undiagnosed due to the insufficient predictive value of questionnaires and limited access to confirmatory video‐polysomnography. We assessed a two‐stage screening strategy combining a brief questionnaire on rapid‐
Caleb A. Massimi +17 more
wiley +1 more source
Chinese expert consensus for neurological monitoring and long-term follow-up in neonatal patients supported on extracorporeal membrane oxygenation (ECMO) (2025): a protocol. [PDF]
Lu M, Li J, Jiang S, Shi J, Helan Y.
europepmc +1 more source
ABSTRACT Objectives We aimed to determine the frequency of subclinical optic nerve (ON) lesions using MRI, optical coherence tomography (OCT), and visual evoked potentials (VEP) in radiologically isolated syndrome (RIS), and to assess their diagnostic and prognostic significance.
Christine Lebrun‐Frenay +13 more
wiley +1 more source
The analysis of competing hypotheses and expert witness testimony: Counteracting adversarial allegiance in witness credibility assessments? [PDF]
Otzipka J, Volbert R.
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
Intraoperative peripheral intravenous complications in adults: a summary of evidence for prevention and management of infiltration/extravasation. [PDF]
Liu H +8 more
europepmc +1 more source
Expert evidence Abstract This thesis deals with the use of expert opinion as evidence in criminal proceedings, the conditions for its admissibility and possible defects of such expert opinions.
Šváb, Martin
core +4 more sources

