Friedreich’s ataxia (FA) is an autosomal recessive disorder caused by mutations in the FXN gene and characterized by cardiomyopathy, gait ataxia and sensory loss, with cardiac complications the main cause of death among patients with FA.
Trigeminal neuralgia (TN) is a chronic disorder caused by the hyperactive functioning of a damaged trigeminal nerve that provokes severe facial pain coming from the trigeminal nerve.
Sania Therapeutics Inc. is setting out its stall at the American Society of Gene & Cell Therapy (ASGCT) conference in Los Angeles this week, after generating proof of concept for its chemogenetics approach to treating motor disorders. The company has engineered adeno-associated viral vectors that can be targeted to specific cell types. It will use these to deliver well-characterized ion channels to dysfunctional motor neurons.
Sironax has described NAD(+) hydrolase SARM1 (SAMD2; MyD88-5) inhibitors reported to be useful for the treatment of amyotrophic lateral sclerosis, Parkinson’s disease, multiple sclerosis, traumatic brain injury, diabetic neuropathy and chemotherapy-induced peripheral neuropathy.
Merck Sharp & Dohme Corp. has disclosed receptor-interacting serine/threonine-protein kinase 1 (RIPK1; RIP-1) inhibitors reported to be useful for the treatment of amyotrophic lateral sclerosis and inflammation.
Diamond Therapeutics Inc. has identified deuterated lisuride analogues acting as 5-HT2A receptor agonists reported to be useful for the treatment of neurological disorders.
Researchers at Mossakowski Medical Research Centre at the Polish Academy of Sciences (PAS) and The University of Warsaw have divulged peptides reported to be useful for the treatment of pain.
By analyzing gene expression patterns in the placenta of nearly 150 pregnancies and comparing them to fetal gene expression in the brain, researchers from the Lieber Institute for Brain Development have gained new insights into the importance of placental tissue in setting the risk trajectory for the development of schizophrenia. The work was published in Nature Communications on May 15, 2023.
N-Methyl-D-aspartate (NMDA) receptors are essential components of the glutamatergic pathways in the central nervous system. NMDA receptors comprise two GluN1 subunits and two GluN2 subunits. Of the latter, the GluN2B subunit is involved in synapse development and plasticity, and cognitive function.