University of California Oakland has described potassium channel subfamily K member 2 (TREK-1; KCNK2) and/or potassium channel subfamily K member 10 (TREK2; KCNK10) activators reported to be useful for the treatment of Caisson disease (decompression sickness), depression, headache, pulmonary hypertension, insomnia, chronic pain, lung and neuronal Injury.
Trace amine-associated receptor 1 (TAAR1) is a member of the G protein-coupled receptor (GPCR) TAAR family, which has been shown to be enriched in the central nervous system and periphery. TAAR1 can couple to diverse G protein subtypes, including Gs, Gq and Gi. Shandong University investigators have recently reported the discovery of a novel TAAR1 agonist as an antipsychotic drug candidate.
Voyager Therapeutics Inc. has announced the selection of a lead development candidate in the GBA1 gene therapy program for the treatment of Parkinson’s disease and other GBA1-mediated diseases under its collaboration with Neurocrine Biosciences Inc.
Preclinical data of rabbits having convulsions has prompted the U.S. FDA to place a clinical hold on Neumora Therapeutics Inc.’s phase I study of NMRA-266 in healthy adults. Neumora said about 30 participants had been dosed so far in the single ascending and multiple ascending dose study, with no evidence of convulsions seen.
Wall Street may not have responded as positively as Ultragenyx Pharmaceutical Inc. would have liked after the firm unveiled new data from the phase I/II study with GTX-102 for the treatment of Angelman syndrome (AS). Patients in expansion cohorts A & B treated with a set dose and regimen of the intrathecally delivered antisense oligonucleotide (ASO) showed rapid and clinically meaningful improvement across multiple domains.
Investors anxious to see data from Marinus Pharmaceuticals Inc.’s phase III Raise study testing intravenous ganaxolone in refractory status epilepticus (RSE) will have to wait a little longer, as the trial failed to meet the predefined criteria for stopping at the interim analysis, sending shares (NASDAQ:MRNS) down nearly 83%.
The U.S. FDA granted Roche Holding AG breakthrough device designation for the Elecsys pTau217 assay that it is developing with Eli Lilly and Co. The test will help with the earlier diagnosis of Alzheimer's disease as it will be able to identify pTau217, a phosphorylated fragment of the protein tau, which is a biomarker that can distinguish the disease from other neurodegenerative disorders.
Laboratory Corp. of America Holdings introduced a glial fibrillary acidic protein blood-based test available commercially in the U.S. for the early detection of neurodegenerative diseases and brain injuries. The test, which the company said was the first of its kind, is designed to assess the presence and progression of Alzheimer’s disease, multiple sclerosis, glioblastoma and traumatic brain injury.
Shandong Ruzhi Biomedical Technology Co. Ltd. has synthesized compounds reported to be useful for the treatment of neurodegeneration, cardiovascular disorders and cerebrovascular disorders.
Researchers from Hefei Industrial Pharmaceutical Institute Co. Ltd. published data detailing the design and biological evaluation of novel NMDAR-GluN2B antagonists as potential candidates for the treatment of ischemic stroke.
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