A phase 3 clinical trial demonstrates the efficacy of olezarsen in reducing plasma triglycerides and acute pancreatitis episodes in patients with familial chylomicronemia syndrome.
Plozasiran, a first-in-class RNA interference therapeutic, reduced triglycerides by 80% and lowered the risk of acute pancreatitis by 83% in patients with familial chylomicronemia syndrome, earning FDA...
The FDA has approved concizumab-mtci, a daily subcutaneous treatment to reduce bleeding for patients with hemophilia who have clotting factor inhibitors.
A specialized inpatient sickle cell disease (SCD) consult service led to improved pain management, shorter hospital stays and expanded use of individualized pain plans for patients with SCD.
Oxalate nephropathy is a commonly underrecognized cause of chronic kidney disease and end-stage kidney disease with multiple etiologies, including primary hyperoxaluria, enteric hyperoxaluria, or ingestion...
A recent cross-sectional, observational study compared the tau spread in people with Down syndrome and autosomal-dominant Alzheimer disease to identify tau-related differences between the two groups.
Researchers examined the epidemiology of hydroxymethylbilane synthase (HMBS) gene defects and activity levels among patients with acute intermittent porphyria to better understand the role of HMBS activity...
