Facioscapulohumeral muscular dystrophy (FSHD) is an autosomal dominant skeletal muscle disorder with a prevalence of approximately 1 in 8,000. The disorder is driven by aberrant expression of double homeodomain protein 4 (DUX4) within the D4Z4 macrosatellite array. Currently, effective treatments for FSHD are lacking. Strategies aimed at reducing DUX4 expression could hold promise as potential therapeutic approaches for FSHD.
Chengdu Kanghong Pharmaceutical Co. Ltd. has disclosed leucine-rich repeat kinase 2 (LRRK2; dardarin) inhibitors reported to be useful for the treatment of cancer, type 1 diabetes, glaucoma, inflammatory bowel disease, multiple sclerosis, neurodegeneration, psoriasis and rheumatoid arthritis, among others.
Nippon Zoki Pharmaceutical Co. Ltd. has discovered alkyne derivatives acting as GABA-A receptor subunit α1β2γ2 positive allosteric modulators. As such, they are believed to be useful for the treatment of sleep disorders.
Orexin OX2 receptor agonists have demonstrated the ability to enhance wakefulness in rodent models, as well as in nonhuman primates and patients with narcolepsy and idiopathic hypersomnia. Based on recent findings, it has been hypothesized that they may also regulate cognition, mood and other neuropsychiatric functions. Furthermore, dysregulated orexin signaling has been reported in patients with major depressive disorder (MDD) with suicide attempts.
Insilico Medicine Cayman Topco and Hygtia Therapeutics Co. Ltd. have entered into an exclusive license and co-development collaboration to advance ISM-8969 worldwide. ISM-8969 is an orally available, brain penetrant NLRP3 inhibitor for CNS disorders.
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.
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