Suppressing Wnt/beta-catenin — a signaling pathway that plays a key role in liver health — significantly reduced liver damage and…
Articles by Marta Figueiredo, PhD
Six months of treatment with Givlaari (givosiran) effectively reduced pain during and between porphyria attacks in people with acute…
People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of…
The first wave of COVID-19Â in Europe severely disrupted access to care and raised stress and anxiety in people with…
Treatment with Panhematin (hemin for injection) is associated with a 78% lower average annual total cost of care…
Excessive levels of estrogen associated with hormone therapy changes and risk factors such as smoking may lead to porphyria…
The U.S. Food and Drug Administration (FDA) has approved Alnylam Pharmaceuticals’ investigational candidate givosiran,  which will be marketed…
The ClpX protein recently has been studied for its role in causing erythropoietic protoporphyria (EPP). In a review study, researchers explored…
Alnylam Pharmaceuticals, after consulting with the U.S. Food and Drug Administration, plans to pursue full approval of its…