Results 91 to 100 of about 815,394 (267)
ABSTRACT Objective To determine the concentration of glial fibrillary acidic protein (GFAP) in cerebrospinal fluid (CSF) and plasma in Alexander disease (AxD) and whether GFAP levels are predictive of disease phenotypes. Methods CSF and plasma were collected (longitudinally when available) from AxD participants and non‐AxD controls.
Amy T. Waldman +9 more
wiley +1 more source
Shengnan Huang,1,2,* Chengzhi Song,3,* Tengyue Zhao,4,* Yefei Yang,4 Zhiwei Yao,4 Shaofeng Duan,5 Wanyi Chen,6 Lixian Li,6 Xiaogang Hu,6 Chunming Li,6 Yurong Hu4 1Academy of Chinese Medical Sciences, Henan University of Chinese ...
Huang S +10 more
doaj
Clinically Relevant Outcome Measures in Women With Adrenoleukodystrophy
ABSTRACT Adrenoleukodystrophy is a rare inherited peroxisomal disease caused by pathogenic variants in the ABCD1 gene located on the X chromosome. Although the most severe central nervous system and adrenal complications typically affect only men with adrenoleukodystrophy, the majority of women develop myeloneuropathy symptoms in adulthood.
Chenwei Yan +3 more
wiley +1 more source
Dosing Optimization of Lamotrigine in Peripregnancy Epilepsy Through PopPK Modelling and Simulation
Jiping Huo,1,2,* Yilin Liu,3,* Jiayu Yang,1,2,* Meng Chen,4,* Li Yang,1 Lijun Wang,5 Dongjie Zhang,1 Tongtong Liu,1 Wanli Gao,5 Haibin Dai,4 Shenghui Mei,1,2 Zhigang Zhao1,2 1Department of Pharmacy, Beijing Tiantan ...
Huo J +11 more
doaj
Remote Assessment of Ataxia Severity in SCA3 Across Multiple Centers and Time Points
ABSTRACT Objective Spinocerebellar ataxia type 3 (SCA3) is a genetically defined ataxia. The Scale for Assessment and Rating of Ataxia (SARA) is a clinician‐reported outcome that measures ataxia severity at a single time point. In its standard application, SARA fails to capture short‐term fluctuations, limiting its sensitivity in trials.
Marcus Grobe‐Einsler +20 more
wiley +1 more source
Xiaohai Chen,* Hualu Wu,* Haoxin Fu, Peiqi Wang, Lu Cao, Ruibin Li, Ruanjuan Zhan, Ren-Ai Xu Department of Pharmacy, The First Affiliated Hospital of Wenzhou Medical University, Wenzhou, Zhejiang, People’s Republic of China*These ...
Chen X +7 more
doaj
ABSTRACT Introduction Progressive Supranuclear Palsy (PSP) is a neurodegenerative ‘tauopathy’ with predominating pathology in the basal ganglia and midbrain. Caudal tau spread frequently implicates the cerebellum; however, the pattern of atrophy remains equivocal.
Chloe Spiegel +8 more
wiley +1 more source
María Roldán Galnares,1 Ramón Morillo-Verdugo,1 María de las Aguas Robustillo-Cortes,1 Manuel Vélez-Díaz-Pallarés,2 María José Company Albir,3 Beatriz Proy Vega,4 Laura Losa López,5 Laura Marín Ventura,6 María Ferris Villanueva,7 Oihana Mora ...
Roldán Galnares M +10 more
doaj
Value of MRI Outcomes for Preventive and Early‐Stage Trials in Spinocerebellar Ataxias 1 and 3
ABSTRACT Objective To examine the value of MRI outcomes as endpoints for preventive and early‐stage trials of two polyglutamine spinocerebellar ataxias (SCAs). Methods A cohort of 100 participants (23 SCA1, 63 SCA3, median Scale for the Assessment and Rating of Ataxia (SARA) score = 5, 42% preataxic, and 14 gene‐negative controls) was scanned at 3T up ...
Thiago J. R. Rezende +26 more
wiley +1 more source

