Sundance Biosciences Inc. has discovered non-receptor tyrosine-protein kinase TYK2 inhibitors potentially useful for the treatment of multiple sclerosis.
Leigh syndrome is a fatal pediatric neurodegenerative disorder caused by mitochondrial dysfunction, most often due to defects in the mitochondrial respiratory chain. The Ndufs4 knockout (Ndufs4 KO) mouse is an established model of the disease, as loss of the NDUFS4 subunit leads to complex I (CI) deficiency and reproduces the neurological decline and pathology seen in affected children. Researchers from The Children’s Hospital of Philadelphia Research Institute and collaborators described how NV-354, a water-soluble prodrug of succinate, may mitigate this mitochondrial dysfunction.
Zincure Corp. has discovered matrix metalloproteinase-9 (MMP-9, gelatinase B) inhibitors reported to be useful for the treatment of epilepsy, stroke, multiple sclerosis, hypoglycemic encephalopathy and traumatic brain injury.
Brain-derived tau, a protein that is exclusive to the brain and detectable in the blood, could serve as an indicator of brain damage after an ischemic stroke. The analysis of this special form of tau has revealed a relationship between high levels of the protein and extensive brain injury, a higher risk of complications, and poorer outcomes.
Latigo Biotherapeutics Inc. has synthesized sodium channel protein type 10 subunit α (SCN10A; Nav1.8) blockers. They are reported to be useful for the treatment of pain, cough and pruritus.
Alexion Pharmaceuticals Inc. has reported complement C1s subcomponent (C1S) inhibitors. They are described as potentially useful for the treatment of amyotrophic lateral sclerosis, rheumatoid arthritis, lupus nephritis and more.
Charcot-Marie-Tooth (CMT) disease is a group of clinically and genetically heterogeneous sensorimotor peripheral neuropathies. It is the most frequent inherited neuromuscular disorder affecting 9.7-82.3 patients per 100,000 individuals. Over 100 genes with all patterns of inheritance have been linked to CMT. These genes encode proteins involved in nerve-specific processes, such as axonal transport, myelination and synaptic transmission, and in general housekeeping pathways. However, the reason underlying why defects in such ubiquitous proteins predominantly affect peripheral nerves remains unclear.
About one-third of patients with major depressive disorder (MDD) are treatment resistant. Ketamine is very effective in treatment-resistant depression, but it is associated with psychotomimetic effects. Metabotropic glutamate mGlu2 and mGlu3 receptors negatively regulate the release of glutamate and are involved in the pathogenesis of depression.
Adolore Biotherapeutics Inc. has announced that the FDA has granted orphan drug designation to the company’s Kv7-activating rdHSV-CA8* gene therapy for treatment of primary and secondary erythromelalgia.
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