Results 281 to 290 of about 2,152,541 (363)
Central Dysmyelination in SSADH‐Deficient Humans and Mice
ABSTRACT Objectives Succinic semialdehyde dehydrogenase deficiency (SSADHD) is an inherited metabolic disorder characterized by an accumulation of γ‐aminobutyric (GABA). In addition to its synaptic role as an inhibitory neurotransmitter, GABA also plays an important role in myelination.
Itay Tokatly Latzer +11 more
wiley +1 more source
Ultrasensitive bioluminescent reporters of protein kinase A based on luciferase activity modulated by phosphorylation (PKA LAMP). [PDF]
Kong Y, Strack S.
europepmc +1 more source
ABSTRACT Objective Neurodegeneration with brain iron accumulation (NBIA) comprises rare genetic disorders characterized by predominantly extrapyramidal symptoms and iron deposition in the basal ganglia. Conventional magnetic resonance imaging (MRI) detects qualitative changes but cannot accurately quantify iron accumulation. Quantitative susceptibility
Özge Uygun +21 more
wiley +1 more source
NEK8, a NIMA-family protein kinase at the core of the ciliary INV complex. [PDF]
Roig J.
europepmc +1 more source
ABSTRACT Objective Recurrent high‐grade gliomas have a poor prognosis and limited therapeutic options. This study aimed to evaluate the safety and efficacy of SYHA1813, a dual inhibitor of VEGFR and CSF1R, in patients with recurrent high‐grade gliomas.
Zhuang Kang +16 more
wiley +1 more source
Dual-specificity mitogen-activated protein kinase kinases can use ADP to phosphorylate MAP kinases invitro. [PDF]
Juyoux P +3 more
europepmc +1 more source
Durability of Response to B‐Cell Maturation Antigen‐Directed mRNA Cell Therapy in Myasthenia Gravis
ABSTRACT Objective We report the 12‐month follow‐up outcomes from a Phase 2 clinical trial (NCT04146051) evaluating Descartes‐08, a BCMA‐directed RNA chimeric antigen receptor T‐cell (rCAR‐T) therapy for refractory generalized myasthenia gravis (MG).
Nizar Chahin +10 more
wiley +1 more source
The prognostic value of mitogen-activated protein kinase kinase in liver hepatocellular carcinoma by bioinformatics. [PDF]
Dong S +4 more
europepmc +1 more source
Assessing a Mitochondrial Disease Treatment via a Novel Statistical Technique for Accelerometer Data
ABSTRACT Objective Therapeutic development for mitochondrial diseases, rare genetic disorders with pathogenic defects of oxidative phosphorylation, is hindered by unsatisfactory outcome measures. To address this problem, we provide the first clinical application of a novel, bias‐adjusted outcome measure of acceleration across a range of subjects ...
Ian W. McKeague +8 more
wiley +1 more source

