Results 261 to 270 of about 159,025 (352)

Early Intervention and Speed-to-Effect in Spinal Muscular Atrophy Type 1 Following Onasemnogene Abeparvovec Gene Replacement Therapy: Results of aPost-Hoc Analysis of Pooled Clinical Study Data [PDF]

W. Toro, Sandra P. Reyna, Shannon Ritter, Anish Patel, Nayla Mumneh, Omar Dabbous   +5 more
openalex   +1 more source

A Novel CYP2E1 Inhibitor, 4‐Methyl‐5‐Acetylthiazole (Q11), Alleviates Obesity Via Modulating Adipose Inflammation and Mitochondrial Dysfunction

Advanced Science, EarlyView.
Obesity involves chronic inflammation and mitochondrial dysfunction. This study identifies cytochrome P450 2E1 (CYP2E1) as a novel inflammatory target in adipose tissue. Its activity increases in obese mice and correlates with inflammation and mitochondrial impairment.
Jinhuan Qiu, Liyuan Gao, Liyang Wang, Xueke Wang, Lin Jia, Mengyan Deng, Liming Tang, Qiang Wen, Na Gao, Haiwei Xu, Hailing Qiao   +10 more
wiley   +1 more source

Real-world multidisciplinary outcomes of onasemnogene abeparvovec monotherapy in patients with spinal muscular atrophy type 1: experience of the French cohort in the first three years of treatment [PDF]

Isabelle Desguerre, Rémi Barrois, Frédérique Audic, Christine Barnérias, B. Chabrol, Jean Baptiste Davion, J. Durigneux, Caroline Espil‐Taris, Marta Gómez‐García de la Banda, Marine Guichard, Arnaud Isapof, Macarena Nougues, Vincent Laugel, Laure Le Goff, Sandra Mercier, Anne Pervillé, Christian Richelme, Marie Thibaud, Catherine Sarret, Cyril Schweitzer, Hervé Testard, Valérie Trommsdorff, Catherine Vanhulle, Ulrike Walther‐Louvier, Cécilia Altuzarra, Mondher Chouchane, Juliette Ropars, Susana Quijano‐Roy, C. Cancès   +28 more
openalex   +1 more source

Mendelian Randomization and Double Machine Learning Modeling Reveal Brain Imaging‐Derived Phenotypes as Functional Contributors to 18 Autoimmune Inflammatory Diseases

Advanced Science, EarlyView.
This schematic integrates the eight statistically significant causal relationships identified between 1,366 brain imaging‐derived phenotypes (IDPs) and 18 autoimmune inflammatory diseases (AIDs). Arrows indicate the direction of causality inferred from bidirectional two‐sample MR analyses.
Jinbin Chen, Xin Wang, Haifeng Ding, Bosheng Zheng, Keni Zeng, Chuying Hu, Jiayi Liu, Xiao Zhu, Haibing Yu   +8 more
wiley   +1 more source

Body Weight and Range of Motion as Predictors of Trunk Asymmetry in Children With Spinal Muscular Atrophy: A Prospective Functional Assessment. [PDF]

Med Sci Monit
Gajewska E, Bieniaszewska A, Sobieska M.
europepmc   +1 more source

Stepwise Regulation of Cellular Oxidative Stress via Conductive‐Piezoelectric Integrated Microstructured Conduits for Enhanced Nerve Regeneration

Advanced Science, EarlyView.
This study presents a conductive‐piezoelectric integrated microstructured conduit for peripheral nerve regeneration. The conduit combines reduced graphene oxide with a piezoelectric nanofiber membrane, enhancing nerve repair through physical guidance, oxidative stress inhibition, and ultrasound‐activated electrical stimulation.
Dong Zhou, Mengxuan Bian, Lingyu Wei, Binghuang Yao, Qi Chen, Lan Xiao, Jing He, Jian Zhang, Yulin Li, Libo Jiang   +9 more
wiley   +1 more source

Development of the "SMA NNE," a short neonatal neurological examination for newborns with spinal muscular atrophy. [PDF]

Eur J Pediatr
Mercuri E, Coratti G, Cutrona C, De Sanctis R, Stanca G, Cicala G, Villa M, Capasso A, Arpaia C, Sbarbati M, Krosschell K, Romeo D, Berti B, Finkel R, Pane M.   +14 more
europepmc   +1 more source

Ultrasound Controlled‐Release Hydrogel Promotes Diabetic Wound Healing via Neuroimmune Modulation and Synergistic ROS Scavenging

Advanced Science, EarlyView.
This study presents an ultrasound‐responsive hydrogel (MCF@CA) that co‐delivers a neuropeptide (CGRP) and a ROS‐scavenging manganese porphyrin to diabetic wounds. The system restores neuro‐immune communication, reprograms macrophages toward an anti‐inflammatory phenotype, and clears excess ROS, thereby accelerating wound closure and promoting mature ...
Mofan Li, Mengxin Wang, Haonan Wang, Yang Sun, Yongyue Zhang, Shuyu Xu, Tianjiao Zhang, Shiti Shama, Xiaolong Liang, Shumin Wang   +9 more
wiley   +1 more source

Course of joint range of motion in children with spinal muscular atrophy receiving disease-modifying treatment. [PDF]

Orphanet J Rare Dis
Oude Lansink ILB, Gorter JW, van der Pol WL, Vd Woude DR, Scheiberlich PJ, van Eijk RPA, Bartels B, Beelen A.   +7 more
europepmc   +1 more source