Charcot-Marie-Tooth disease type 2E (CMT2E) is a slow and progressive neuropathy characterized by axonal dysfunction. Its clinical phenotype includes muscle weakness and atrophy, sensory loss and reduced nerve conduction velocity, among others.

At this week’s Muscular Dystrophy Association Clinical and Scientific Conference in Dallas, researchers from Suzhou Genassist Therapeutics Co. Ltd. presented preclinical data for GEN-6050X (ss.AAV9.oTAM and ss.AAV9.hE50-sgRNA).

F. Hoffmann-La Roche Ltd. and Hoffmann-La Roche Inc. have identified new benzimidazoles acting as serine/threonine-protein kinase SIK inhibitors reported to be useful for the treatment of rheumatoid arthritis, metabolic dysfunction-associated steatohepatitis (MASH), giant cell arteritis, primary sclerosing cholangitis, inflammatory bowel disease, atherosclerosis, type 2 diabetes and glomerulonephritis, among others.

Bristol Myers Squibb Co. has patented new protein-arginine deiminase type-4 (PADI4; PAD4) inhibitors reported to be useful for the treatment of rheumatoid arthritis, among others.

Due to the widespread presence of PDE7 in various tissues, the development of PDE7 inhibitors has been limited by potential adverse side effects.