Bleeding of unknown cause is a group of rare disorders that are still difficult to accurately diagnose. A case report on a patient with hematoma in the perineal region after her first delivery was presented.
Palo Alto, Calif.-based Bridgebio Pharma Inc. will hand over development and sales of its rare bone growth disorder therapy, infigratinib, in Japan to Kyowa Kirin Co. Ltd. under its latest exclusive licensing deal.
Vandria SA has been awarded two grants totaling €3.8M (US$4.1M) from Innosuisse and Eurostars to support its two lead drug candidates addressing CNS and muscle diseases, respectively.
Muna Therapeutics ApS has disclosed new triggering receptor expressed on myeloid cells 2 (TREM2) agonists reported to be useful for the treatment of osteoporosis, rheumatoid arthritis, systemic lupus erythematosus, type 2 diabetes, obesity, metabolic dysfunction-associated steatotic liver disease, neurodegenerative and inflammatory bowel disease, among others.
Previous research has shown that the three isoforms of transforming growth factor β (TGF-β) have distinct cellular expression patterns, with TGF-β2 and TGF-β3 expression being specifically elevated in human fibrotic lung and liver tissue. Since pan-TGF-β inhibition had proven to be toxic for chronic use, in recent work, researchers from Genentech Inc. aimed to assess whether specifically targeting one of the TGF-β isoforms could represent a novel therapeutic option for patients with chronic fibrotic disorders.
Japanese researchers have presented data from the biggest Asian genome-wide association study (GWAS) regarding susceptibility loci for systemic sclerosis (SSc), comprising a total of 1,428 cases and 112,599 controls and with an imputation reference panel containing more than 3,000 Japanese whole-genome sequencing data.
China Pharmaceutical University has synthesized kaurane tetracyclic diterpenoid derivatives acting as NLRP3 inflammasome inhibitors reported to be useful for the treatment of arthritis and skin disorders.
Edgewise Therapeutics Inc. priced an underwritten offering of 21.8 million shares at $11 each as it looks for about $240 million in gross proceeds to develop its Duchenne and Becker muscular dystrophies treatment. The offering propelled the company’s stock (NASDAQ:EWTX) Jan. 19 to close 34.5% higher at $13.04 each, their highest valuation in the past 12 months.
Researchers at F. Hoffmann-La Roche Ltd. and Hoffmann-La Roche Inc. have described imidazo[4,5-b]pyridine and pyrazolo[1,5-a]pyrimidine derivatives acting as serine/threonine-protein kinase SIK inhibitors reported to be useful for the treatment of juvenile idiopathic arthritis, nonalcoholic steatohepatitis (NASH), primary sclerosing cholangitis, inflammatory bowel disease, atherosclerosis, type 2 diabetes and glomerulonephritis, among others.
The stress-responsive protein PPP1R15A (protein phosphatase 1, regulatory subunit 15 A) promotes the unfolded protein response and restores protein homeostasis.
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