The KCNC1 gene encodes the voltage-gated potassium channel subunit KV3.1, and variants in this gene have been previously linked to developmental and epileptic encephalopathy (DEE).
Xalud Therapeutics Inc. has described conjugates comprising a sugar moiety covalently bonded to a DNA-interacting moiety through a linker reported to be useful for the treatment of pain, inflammation and autoimmune diseases, among others.
Cypralis Ltd. has divulged macrocyclic compounds acting as peptidyl-prolyl cis-trans isomerase A (PPIA; cyclophilin A; CYPA) and/or cyclophilin D inhibitors reported to be useful for the treatment of multiple sclerosis and epilepsy.
Researchers from Gilgamesh Pharmaceuticals Inc. and affiliated organizations have presented the discovery and preclinical evaluation of GM-3009, a novel noribogaine analogue being developed for the treatment of opioid use disorder (OUD).
The term cerebral small vessel disease (SVD) refers to the sum of all pathological processes affecting the small vessels of the brain. Although SVD has been recognized as a leading cause of age-related cognitive decline, its underlying mechanisms remain unknown. Moreover, therapeutic options for SVD patients are still limited, primarily because relevant disease models that can guide target identification and drug discovery are lacking.
Researchers at Tufts University and University of Houston System have identified receptor-interacting serine/threonine-protein kinase 2 (RIPK2; RIP-2) and/or nucleotide-binding oligomerization domain-containing protein 2 (NOD2) inhibitors reported to be useful for the treatment of multiple sclerosis.
Researchers from West China Hospital of Sichuan University presented data from a study that aimed to investigate the associations between serum cystatin C (CysC) levels and the progression and survival of patients with amyotrophic lateral sclerosis (ALS).
Vandria SA has announced a series A financing round raising $20.6 million (CHF18 million) to help advance its pipeline of first-in-class small-molecule mitophagy inducers.