It has been previously demonstrated that noise exposure leads to activation of the mitogen-activated protein kinase (MAPK) pathway, and that inhibition of this pathway protects from hearing loss. Tizaterkib (formerly AZD-0364) is a novel, highly selective, orally bioavailable ERK1/2 inhibitor that is currently in early clinical development for the treatment of cancer. In recent work, investigators from Creighton University aimed to assess the potential of tizaterkib for the treatment of noise-induced hearing loss (NIHL).

Bleeding of unknown cause (BUC) is a diagnosis of exclusion, and it is common for these patients to have congenital platelet function disorders. Whole-exome sequencing may help reach a more accurate diagnosis in these cases.

Agonists of the glucagon-like peptide-1 receptor (GLP-1RAs) have emerged as effective treatments for obesity, but have a negative impact on lean mass during weight loss.

At the ongoing WORLDSymposium meeting in San Diego, Gain Therapeutics Inc. presented preclinical data on the company’s GCase enhancer GT-02287 as a potential approach to rescue motor function in Parkinson’s disease (PD).

Researchers from Scynexis Inc. have recently presented details on the preclinical characterization of SCY-247, a second-generation triterpenoid with glucan synthase inhibitor activity and intended to be used for the treatment of invasive mucormycosis.

Hemoglobinopathies affect 7% of the global population, and sickle cell disease (SCD) is a common form affecting 300,000 newborns per year.