Acute rheumatic fever (ARF) is an autoimmune disorder resulting from Group A Streptococcus (GAS) pharyngitis or impetigo in children and adolescents, which may evolve to rheumatic heart disease (RHD) with persistent cardiac valve damage. RHD causes substantial mortality and morbidity globally, predominantly among socioeconomically disadvantaged populations, with an interplay of social determinants of health and genetic factors determining overall risk. ARF diagnosis is based on a constellation of clinical and laboratory features as defined by the 2015 Jones Criteria, although advances in molecular point-of-care testing and the ongoing search for ARF biomarkers offer the potential to revolutionise diagnostics. There are persistent gaps in ARF pathophysiology with little progress in therapeutics over the last several years. The greater focus towards primordial, primary, and secondary prevention such as advances in GAS vaccine development, innovations in digital health technology, improved antibiotic formulations for secondary prevention, and decentralised programmatic implementation to improve health-care delivery offer feasible solutions towards reducing future ARF burden globally.

Currently, no worldwide approved therapies exist for adults with neurofibromatosis type 1 (NF1) and symptomatic, inoperable plexiform neurofibromas. The KOMET study aimed to evaluate selumetinib (ARRY-142886, AZD6244) efficacy and safety in this population.