Fragile X syndrome (FXS) is the most common inherited form of intellectual disability and a leading monogenic cause of autism, yet effective treatments remain elusive. Previous work showed that N-methyl-D-aspartate receptors (NMDARs) play a prominent pathophysiological role in FXS and other neurodevelopmental disorders.
Researchers from General Hospital of Tianjin Medical University presented data from a study that aimed to explore the role of NIMA-related kinase 2 (NEK2) in regulating B-cell immunity in autoimmune diseases. It was demonstrated that NEK2 is highly expressed in patients with multiple sclerosis.
Transthera Biosciences Co. Ltd. has divulged NLRP3 inflammasome inhibitors reported to be useful for the treatment of autoimmune diseases and inflammatory disorders.
Merck Sharp & Dohme LLC has synthesized viral replication inhibitors reported to be useful for the treatment of respiratory syncytial virus and metapneumovirus infections.
Prelude Therapeutics Inc. has disclosed phosphatidylinositol 3-kinase α (PI3Kα) (H1047R mutant) inhibitors reported to be useful for the treatment of cancer, PIK3CA-related overgrowth spectrum and more.
THB-335 is an oral inhibitor of mast/stem cell growth factor receptor Kit (KIT), which regulates the activation and migration of mast cells, making it a relevant therapeutic target for inflammatory and allergic processes.
Exposure to high-dose radiation can result in gastrointestinal (GI) acute radiation syndrome, which is characterized by disruption of the mucosal layer, intestinal epithelial barrier dysfunction, and abnormal inflammatory responses. Currently, there are no FDA-approved therapies for the treatment radiation-induced GI injury.
Esophageal cancer, particularly esophageal squamous cell carcinoma (ESCC), is one of the most prevalent digestive malignancies, with a 5-year survival rate of only 20%. Although some cancer immunotherapies based on immune checkpoint inhibitors (ICIs) have shown promise for ESCC, only a minority of patients actually benefit from ICI therapy.
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