The U.S. Food and Drug Administration approved inebilizumab-cdon as the first and only treatment for IgG4-related disease, a rare, chronic immune-mediated inflammatory condition that affects multiple organs. The approval was based on results...

A recent poster presented at the 2025 American College of Cardiology's Annual Scientific Session evaluated surgical outcomes in patients with homozygous familial hypercholesterolemia (HoFH) undergoing aortic valve replacement (AVR). Researchers analyzed data from 134...

Although most children adhere well to daily recombinant human growth hormone (rhGH) therapy, a significant portion experience therapy-related stress, with discomfort among peers due to the chronic treatment a major contributing factor, according...

While sponsored genetic testing panels improve access to molecular diagnosis for inherited retinal diseases (IRD), significant challenges remain, including varying positivity rates between providers, discrepancies in variant classification (especially for variants of unknown...