The U.S. Food and Drug Administration (FDA) granted orphan drug designation to ARO-ANG3 (Arrowhead Pharmaceuticals) for the treatment of homozygous familial hypercholesterolemia (HoFH).
Dennis Jackman, senior vice-president of public affairs at CSL Behring discusses some of the barriers to recruiting participants to be in clinical trials for rare diseases.
The Food and Drug Administration (FDA) granted orphan drug designation to LM-030, an investigational therapy licensed from Novartis and ready to enter into pivotal clinical trials for the treatment of Netherton Syndrome.
The FDA has granted orphan drug status and a rare pediatric disease designation to Denali Therapeutics pipeline candidate, DNL310.
Malissa J. Wood, MD at Harvard Medical School discusses the iSCAD Registry, which develops and maintains an independent, multi-center data repository to advance the pace and breadth of SCAD research around the world. Spontaneous coronary artery dissection (SCAD) is a rare condition and occurs when a tear forms in one of the blood vessels in the heart.
The process for researching and developing new medicines is notoriously time-consuming, complicated and costly – and even more so when it comes to therapies for rare diseases.