Beigene Co. Ltd. has identified 3, 4-dihydroisoquinolin-1 (2h)-ones derivatives acting as stimulator of interferon genes protein (STING; TMEM173) antagonists reported to be useful for the treatment of systemic lupus erythematosus (SLE).
Researchers from Ermium Therapeutics SAS have presented preclinical data for the novel chemokine CXCR4 receptor ligand ER-000145, being developed for the treatment of type 1 interferonopathies, such as systemic lupus erythematosus (SLE).
Researchers from the Institute of Translational Genomics at Helmholtz Munich have described a genetic overlap between type 2 diabetes (T2D), a disease that is also associated with obesity, and osteoarthritis, a degeneration of the joints that worsens with age and coincides in the factor risk of being overweight. The researchers used genetic data, multiomics and functional analysis of the tissues T2D and osteoarthritis express to identify which genes were associated and correlated with both diseases. They published their results on July 10, 2023, in The American Journal of Human Genetics.
Researchers from Anhui Medical University presented the discovery and preclinical evaluation of new vascular endothelial growth factor receptor 2 (VEGFR-2) inhibitors as potential therapeutic candidates for rheumatoid arthritis.
After nearly 10 years in clinical development and just a few months after an EMA rejection, Ipsen SA will be making its case June 28 before a U.S. FDA advisory committee for its ultra-rare bone disease drug, palovarotene.
Sarepta Therapeutics Inc. has set the wholesale acquisition cost of the first gene transfer therapy for ambulatory patients with Duchenne muscular dystrophy at $3.2 million, making it one of the most expensive gene therapies. The company said the gross-to-net price for Elevidys (delandistrogene moxeparvovec) will be in the mid-20% range, which, suggests Mizuho Group analyst Uy Ear, would put the price at about $2.4 million.
Sarepta Therapeutics Inc. is set to introduce the first gene transfer therapy for ambulatory patients diagnosed with Duchenne muscular dystrophy (DMD), after the U.S. FDA granted accelerated approval to SRP-9001 (delandistrogene moxeparvovec). Branded Elevidys, the therapy marks Sarepta’s fourth approved treatment for DMD and the first to offer patients a one-time treatment option.
Researchers from the Norwegian University of Science and Technology (NTNU) reported the discovery of novel colony-stimulating factor 1 receptor (CSF-1R) inhibitors as potential candidates for the treatment of osteoporosis.
It has been previously demonstrated that in zebrafish, the activity of UXS1, a gene that encodes UDP-glucuronate decarboxylase 1, is essential for production and organization of skeletal extracellular matrix. Now, researchers from the University of Oslo and affiliated organizations have identified a novel pathogenic variant in UXS1.
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