NLS Pharmaceutics Ltd., in collaboration with Aexon Labs Inc., has announced new preclinical findings on AEX-2, a non-sulfonamide dual orexin receptor agonist (DOXA) with potential for narcolepsy and related neurological disorders.
The FKBP5 gene encodes FKBP prolyl isomerase 5, a co-chaperone that modulates glucocorticoid signaling and that is expressed in T cells, neurons and in microglial cells in the central nervous system (CNS). The role of FKBP5 in the dysregulation of myeloid cells in the pathogenesis of multiple sclerosis was investigated in a murine model of experimental autoimmune encephalomyelitis.
A recent study by researchers from Nanyang Technological University identified Fanconi anemia complementation group M (FANCM) as a crucial regulator of alternative lengthening of telomere (ALT), aiming to develop new antisense oligonucleotides (ASOs) to suppress its function.
Family with sequence similarity 19 member A5 (FAM19A5) is a secretory protein highly expressed in the brain that regulates synapse dynamics through its interaction with leucine-rich repeat-containing 4B (LRRC4B). Noise exposure is one of the most significant causes of synapse loss between inner hair cells and auditory nerve fibers, leading to a decline in hearing sensitivity.
Researchers from Cedars-Sinai Medical Center presented the preclinical efficacy of KROS-401, an IL-4/IL-13 blocking peptide that effectively reprograms macrophages in glioma models.
Guangdong Fapon Biopharma Inc. has obtained IND clearance from the FDA for FP-008, its first-in-class immunocytokine for patients with solid tumors refractory to anti-PD-1 therapy.
Too much of a good thing, it turns out, is a concept that applies to oxygen. And researchers at the University of California at San Francisco are working on a small molecule, Hypoxystat, that can lower tissue oxygen levels and prevent damage when oxygen levels are too high. When administered to mice with the rare mitochondrial disorder Leigh syndrome, the molecule more than tripled their average lifespan.
Whole genome sequencing has substantially accelerated the pace of discovery of genes that cause rare diseases, but while this has brought the diagnostic odyssey of some patients to a conclusion, 50% to 80% remain undiagnosed after initial analysis. Researchers in the U.K. have now developed a new framework for analyzing sequence data at a cohort level. Applying this method to almost 35,000 undiagnosed rare disease patients led to the identification of 141 new disease-gene associations.
Shanghai Institute of Materia Medica of the Chinese Academy of Sciences and Zhejiang University have jointly discovered N2-3-fluoro-5-substituted phenyl-2-aminopyrimidine derivatives.
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