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
Perioperative management and challenges of robotic-assisted surgery: a narrative review. [PDF]
Xu Y, Wang X.
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
Electrical impedance tomography assessment of bronchoalveolar lavage-associated lung derecruitment and its related factors: a single-center retrospective study. [PDF]
Yang A +9 more
europepmc +1 more source
This present volume contains the text of all contributions (oral and posters), except for the four invited papers, which were presented at the 3rd International Symposium on High Pressure Chemical Engineering on October 7-9, 1996.
Trepp, Ch. +2 more
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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
Characterisation and management of therapy-interrupting system errors associated with the use of the HomeChoice cycler for automated peritoneal dialysis. [PDF]
Albert A +5 more
europepmc +1 more source
Digital Cognitive Phenotyping for Differential Diagnosis and Monitoring in Neurological Conditions
ABSTRACT Objective To assess the utility, accessibility, and equivalence to supervised scales of online cognitive assessment in older individuals with cognitive impairment. Methods Patients with Alzheimer's disease (AD, n = 31), idiopathic normal pressure hydrocephalus (iNPH, n = 26), and traumatic brain injury (TBI, n = 23) completed online cognitive ...
Martina Del Giovane +10 more
wiley +1 more source
Low-Temperature Thermomechanical Consolidation of Aluminosilicate Sorbents Impregnated with Model Oil-Containing Radioactive Waste. [PDF]
Koyanbayev Y +8 more
europepmc +1 more source
Pricing Excess-of-loss Reinsurance Contracts Against Catastrophic Loss [PDF]
This paper develops a pricing methodology and pricing estimates for the proposed Federal excess-of- loss (XOL) catastrophe reinsurance contracts.
J. David Cummins +2 more
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