Hepcidin deficiency in hereditary hemochromatosis (HH) leads to increased absorption of dietary iron and thus iron overload. Rusfertide is a hepcidin mimetic peptide that has shown efficacy at reducing the need for therapeutic maintenance phlebotomy in patients with HH. Researchers aimed to evaluate the benefits of cotreatment with a hepcidin mimetic peptide plus the rusfertide analogue PN-23114 (Protagonist Therapeutics Inc.) at 7.5 mg/kg t.i.w. and phlebotomy in a murine model of HH.

Investigators from Cerevance Inc. have reported the discovery and preclinical characterization of a novel tandem pore domain halothane-inhibited K+ channel 1 (THIK-1) inhibitor, C-101248, being developed for the treatment of neuroinflammation in Alzheimer’s disease (AD). NETSseq and histological analysis revealed that THIK-1 expression was up-regulated in microglia from different cortical regions of AD donors compared with aged matched nondemented control brains.

The archetypal UbiB protein COQ8 has human homologues COQ8A and COQ8B, both with well-established connection to human disease, with inactivating mutations in COQ8A resulting in autosomal recessive cerebellar ataxia. Researchers from the University of Wisconsin-Madison and affiliated organizations have now recently reported the discovery of small-molecule inhibitors of COQ8A.