Results 151 to 160 of about 95,633 (353)
Early exposure to environmental toxin contributes to neuronal vulnerability and axonal pathology in a model of familial ALS [PDF]
Adult onset amyotrophic lateral sclerosis (ALS) arises due to progressive and irreversible functional deficits to the central nervous system, specifically the loss of motor neurons.
Christopher A. Shaw, Grace Lee
core +1 more source
Impact of Rapid Exome Sequencing on Pediatric Patients With Cardiomyopathy and Acute Heart Failure
ABSTRACT Few studies describe the impact of rapid exome sequencing (ES) on pediatric cardiomyopathy in urgent clinical settings. Here, we retrospectively report the impact of rapid singleton ES in pediatric patients presented with acute heart failure and isolated cardiomyopathy or myocarditis, between 2021 and 2023 at a single tertiary care center.
Tameemi Abdalla Moady +10 more
wiley +1 more source
The dose response effect of long-acting nondepolarizing neuromuscular blocking agents in children [PDF]
Nishan G. Goudsouzian +3 more
openalex +1 more source
Objective Myotonic dystrophy type 1 (DM1) is a highly variable, multisystemic genetic disorder caused by a CTG repeat expansion in the 3′ untranslated region of DMPK. Toxicity is exerted by repeat‐containing DMPK transcripts that sequester muscleblind‐like (MBNL) proteins and lead to deleterious yet predictable changes in alternative splicing.
Samuel T. Carrell +3 more
wiley +1 more source
Objective An enduring puzzle in many inherited neurological disorders is the late onset of symptoms despite expression of function‐impairing mutant protein early in life. We examined the basis for onset of impairment in spinocerebellar ataxia type 6 (SCA6), a canonical late‐onset neurodegenerative ataxia which results from a polyglutamine expansion in ...
Haoran Huang +10 more
wiley +1 more source
Objective Biomarkers with clear contexts of use are important tools for amyotrophic lateral sclerosis (ALS) therapy development. Understanding their longitudinal trajectory in the untreated state is key to their use as potential markers of pharmacodynamic response.
Oleksandr Dergai +16 more
wiley +1 more source
Implantable Drug Delivery Systems for Skeletal Muscles and Eyes
This review highlights the different types of recent implantable drug delivery systems (IDDS) fabricated for a use with skeletal muscles, and with eyes. It presents the developments already made and the current research directions, showing the evolution of IDDS and their great diversity.
Serge Ostrovidov +8 more
wiley +1 more source
Micro/Nanorobot for Drug Delivery—A Review of Material Selections
This review explores material strategies for biomedical micro/nanorobots, emphasizing their roles in propulsion, navigation, drug delivery, and biodegradability. It highlights advancements in metallic, polymeric, and hybrid materials, while addressing clinical translation challenges such as toxicity, immune response, and manufacturability.
Xiaozhuo Wu, Bingyun Li, Malcolm Xing
wiley +1 more source

