Duchenne muscular dystrophy is a severe and progressive disorder caused by mutations in the dystrophin (DMD) gene that lead to malfunction or absence of dystrophin. This protein stabilizes the sarcolemma and protects muscle cells during contraction.
The Japanese government is throwing full weight behind the Bioeconomy Strategy initiative to position Japan’s biopharmaceutical industry as both a key driver of economic growth and global drug discovery hub.
Centessa Pharmaceuticals plc recently detailed the discovery and preclinical characterization of a novel potent and selective orexin OX2 receptor agonist, ORX-142.
It was hypothesized that the MEK1/2 inhibitor ATR-002 could reduce inflammation and clear Staphylococcus aureus infection during cystic fibrosis, thus potentially showing a dual effect.
Khalifa University and The University of Queensland presented preclinical data for a novel acid-sensing ion channel 1 (ASIC1) inhibitor, Hi1a, evaluated in models of multiple sclerosis (MS).
The signaling of TNF receptor (TNFR) superfamily member 4 (OX40) and its ligand (OX40L) plays a crucial role in the development of immunological and inflammatory disorders due to triggering a subset of T-cell responses.
As in other muco-obstructive diseases, the airways in cystic fibrosis (CF) are characterized by goblet cell and glandular hyperplasia, with overproduction of mucins MUC5 and MUC5AC, resulting in viscous mucus, respiratory blockade and recurrent infections and inflammation.
At this week’s American Society for Radiation Oncology meeting, scientists from The University of Texas MD Anderson Cancer Center reported the discovery and preclinical evaluation of CD47-LLO, a novel microbial-inspired antibody-drug conjugate (ADC) for the treatment of cancer.
The development of cystic fibrosis transmembrane conductance regulator (CFTR) modulators has significantly improved the therapeutic scenario for CF patients in the past decade. However, around 10% of patients harboring nonsense and splice-site mutations are nonresponsive to CFTR modulators.
Inmagene LLC presented preclinical data for the novel noncovalent reversible Bruton tyrosine kinase (BTK) inhibitor IMG-004, which is in phase I development for autoimmune diseases.
RSS
