Guillain-Barré syndrome (GBS) is an immune-driven inflammatory disorder of the peripheral nervous system characterized by muscle weakness and paralysis. Despite treatment options, GBS stays severe, with a mortality rate of 3%-10%. The mechanisms behind GBS are poorly understood and new therapeutic options are needed.
Aminoglycoside antibiotics are essential for treating some severe bacterial infections but are notorious for causing irreversible hearing loss in 20%-47% of patients.
The new gene therapy aims to address the root cause of prion disease by using the CHARM epigenetic editing platform from the Whitehead Institute to target and silence the gene that codes for the disease-causing protein.
Shenzhen Hightide Biotechnology Ltd. has divulged prodrugs and derivatives of 2,4-dinitro phenol reported to be useful for the treatment of obesity, diabetes, liver disease, heart disease, renal failure, neurodegenerative diseases and aging, among others.
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.
Shanghai Pharmaceuticals Holding Co. Ltd. has disclosed aza-ergoline derivatives acting as dopamine D2 receptor and/or D3 receptor agonists reported to be useful for the treatment of attention deficit hyperactivity disorder (ADHD), depression, erectile dysfunction, substance abuse and dependence, restless legs syndrome, schizophrenia, Parkinson’s and Alzheimer’s disease, among others.
Bridgene Biosciences Inc. has signed a strategic collaboration and licensing agreement with Takeda Pharmaceutical Co. Ltd. to discover small-molecule drug candidates against targets in immunology and neurology.
Researchers from Centro de Investigación Biomédica en Red sobre Enfermedades Neurodegenerativas (CIBERNED), Miramoon Pharma SL and affiliated organizations published the preclinical characterization of FKBP12 ligand MP-010 and evaluated its efficacy in models of amyotrophic lateral sclerosis.
Epstein-Barr virus (EBV) infection is a recognized risk factor that is now regarded as a prerequisite for the development of multiple sclerosis (MS). Recently, significant advances have been made in clarifying the precise mechanism by which EBV leads to the pathogenic features of MS. Now, a new study may have tied up more loose ends. Researchers from the University of Helsinki have mapped the immune landscape of deep cervical lymph nodes (dCLN) in patients recently diagnosed with MS.