BLR Bio LLC announced that the U.S. FDA has granted orphan drug designation to BLR-200, the company’s investigational therapy for the treatment of systemic sclerosis (SSc).
Scientists at Ez Bioxcel Solutions Pvt Ltd., Invea Therapeutics Inc. and Inveniai LLC have identified apoptosis-associated speck-like protein containing a CARD (PYCARD; ASC) inhibitors reported to be useful for the treatment of primary sclerosing cholangitis, arthritis and inflammatory disorders.
Researchers from Catholic University of Korea published data from a study that investigated the effect of miRNA-21a-5p on fibrosis development in systemic sclerosis (SSc). With the aim of assessing the pathological impact of miRNA-21a-5p on skin and lung fibrosis in vivo, a bleomycin-induced SSc murine model was developed, and the mice were hydrodynamically injected with plasmids containing pre-miRNA-21a-5p or anti-miRNA-21a-5p.
About 90% of patients with multiple myeloma (MM) develop severe bone disease, known as myeloma bone disease (MBD). This occurs due to the ability of MM cells to disrupt bone homeostasis, leading to excessive bone resorption. Some current treatments are effective for treating MBD, but they are associated with undesired adverse events.
Genescience Pharmaceuticals Co. Ltd. has divulged MAP kinase-activated protein kinase 2 (MAPKAPK2; MK2) inhibitors reported to be useful for the treatment of rheumatoid arthritis and radiographic axial spondyloarthritis (ankylosing spondylitis).
Recent genome-wide association studies identified an association between low bone mineral density (BMD) and a single-nucleotide polymorphism (SNP) at the MALAT1 locus, but there is no functional evidence on the role of MALAT1 alterations in BMD or osteoporosis. Hence, scientists at MD Anderson Cancer Center aimed to assess the functional role of MALAT1 alterations in low BMD and osteoporosis.
Sudo Biosciences Ltd. has divulged non-receptor tyrosine-protein kinase TYK2 inhibitors reported to be useful for the treatment of scleroderma, radiographic ankylosing spondylitis, type 1 diabetes, idiopathic pulmonary fibrosis, multiple sclerosis, psoriasis, rheumatoid arthritis, Sjögren’s syndrome and systemic lupus erythematosus, among others.
Mutations in the gene encoding calpain-3, CAPN3, are causative for autosomal recessive limb-girdle muscular dystrophy-1 (LGMDR1), a type of muscular dystrophy characterized by muscle weakening around the shoulders and hips for which there is currently no treatment.
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