Lysomal acid lipase (LAL) deficiency is a rare metabolic disorder that also causes liver inflammation. A paper recently published by Medical University of Graz scientists presents a promising ...

Amsterdam-headquartered firm uniQure developed the drug for a small subset of patients with the rare familial lipoprotein lipase deficiency (LPLD), and gained a five year marketing authorisation ...

UniQure's Glybera for lipoprotein lipase deficiency was approved a decade ago, but was pulled off the market a few years later on low demand and questions about its effectiveness. Orchard ...