Netherton syndrome (NS) is a rare genetic disease caused by loss of functional lympho-epithelial Kazal-type-related inhibitor (LEKTI, SPINK5). It was hypothesized that small-molecule inhibitors of KLK5 could replace deficient LEKTI in NS.

The American Society of Clinical Oncology (ASCO) begins its 2024 annual meeting at the cavernous and labyrinthine McCormick Place convention center in Chicago Friday, May 31. It’s one of the world’s largest cancer research conferences and can be daunting to follow.  More than 400 organizations will participate this year, with about 200 sessions ready to convene. The vast majority of the 5,000 abstracts that cover all aspects of cancer treatment have already been released, and they will be scrutinized by the more than 40,000 attendees from around the world.

Acute respiratory distress syndrome (ARDS) is a multifactorial disease, the pathogenesis of which involves environmental exposure and genetic predisposition.

Since utrophin compensates for lack of dystrophin in mdx mice, which results in a milder phenotype of muscular dystrophy compared to humans, the mdx/utrn-/- mouse has been developed to mimic early onset of muscle dystrophy, severe muscle weakness and premature death.

Researchers from the University of Colorado presented preclinical data for the myeloid leukemia cell differentiation protein Mcl-1 inhibitor S-64315, currently in phase I clinical development for the treatment of hematologic cancers.

The cannabinoid CB1 receptor inverse agonist INV-202 (monlunabant) is in clinical studies at Inversago Pharma Inc. as a potentially first-in-class drug for metabolic disorders, including diabetic kidney disease and obesity.