Results 51 to 60 of about 257,184 (268)
Annals of Clinical and Translational Neurology, EarlyView.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
Annals of Clinical and Translational Neurology, EarlyView.ABSTRACT Background X‐linked adrenoleukodystrophy (X‐ALD) is a neurometabolic disorder caused by pathogenic variants in ABCD1, leading to slowly progressive spinal cord disease in nearly all affected men. Sensitive biomarkers to quantify disease severity and predict progression are needed for clinical care and trial design.
Eda G. Kabak +4 more
wiley +1 more source
Criminocorpus, 2019 In Italy at the end of the seventies, many artists are questioned on stage, in violent forms which range from invective to political trial, including shooting, throwing stones or even Molotov cocktails.
Céline Pruvost
doaj +1 more source
Onasemnogene Abeparvovec in Patients With SMA: Interim Results of the RESTORE Registry in Japan
Annals of Clinical and Translational Neurology, EarlyView.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
Distinct Systemic Sclerosis Phenotypes Related to Ethnicity: An Opportunity to Personalize Care?
Arthritis Care &Research, EarlyView.Objective The objective is to describe and compare demographic, clinical, and serological characteristics of patients with systemic sclerosis (SSc) according to ethnic background. Methods Participants enrolled in the Canadian Scleroderma Research Group cohort who self‐identified to a single ethnicity group were included.
Danick Goulet +11 more
wiley +1 more source
Rock and Roll Styles and Genres in Poland (1957–1973)
Musicology Today, 2016 This paper describes the process of establishing rock and roll styles and genres (as defined by Allan F. Moore) in Polish musical culture. My Ph.D. research has revealed three phases in this process. Phase 1: imitation (1957-1962), phase 2: Polonisation (
Gradowski Mariusz
doaj +1 more source
Frontiers in Microbiology, 2018 Schizophyllum commune is a filamentous basidiomycete causing white-rot in many wood species with the help of a broad range of enzymes including multicopper oxidases such as laccases and laccase-like oxidases. Since these enzymes exhibit a broad substrate
Julia Kirtzel +8 more
doaj +1 more source
Special Issue on Fatigue and Fracture of Non-Metallic Materials and Structures
Applied Sciences, 2020 This Special Issue covers the broad topic of structural integrity of non-metallic materials, and it is concerned with the modelling, assessment and reliability of structural elements of any scale. In particular, the articles being contained in this issue
Andrea Spagnoli
doaj +1 more source
Multimodal Data‐Driven Microstructure Characterization
Advanced Engineering Materials, EarlyView.A self‐consistent autonomous workflow for EBSP‐based microstructure segmentation by integrating PCA, GMM clustering, and cNMF with information‐theoretic parameter selection, requiring no user input. An optimal ROI size related to characteristic grain size is identified.
Qi Zhang +4 more
wiley +1 more source
A Practical Noise2Noise Denoising Pipeline for High‐Throughput Raman Spectroscopy
Advanced Engineering Materials, EarlyView.A lightweight and reproducible denoising pipeline for high‐throughput Raman spectroscopy is introduced, based on a 1D convolutional autoencoder trained with a Noise2Noise strategy. Using only repeated short‐exposure acquisitions, the method suppresses stochastic noise without reference spectra, enabling reliable spectral reconstruction while preserving
David Martin‐Calle +5 more
wiley +1 more source