by Peter Ciszewski | Nov 29, 2019
New published data shows that the monoclonal antibody combination therapy, REGN-EB3 developed by Regeneron Pharmaceuticals, and the monoclonal antibody Mab114 developed by the National Institutes of Health and licensed by Ridgeback Biotherapeutics, are superior to...
by Peter Ciszewski | Nov 27, 2019
Luke Rosen, founder of KIF1A.org talks about the origins if this rare disease patient organization to help collect data on children with KIF1A associated neurological disorder (KAND). KAND is due to a mutation in the KIF1A gene. Defects in that gene lead to a...
by Peter Ciszewski | Nov 25, 2019
The U.S. Food and Drug Administration (FDA) has approved Oxbryta (voxelotor) to treat persons, 12 years and older, with sickle cell disease. Voxelotor blocks deoxygenated sickle hemoglobin polymerization and thereby inhibits red blood cell for ‘sickling’. Sickle cell...
by Peter Ciszewski | Nov 25, 2019
Bobby Gasper, MD, Chief Scientific Officer of Orchard Therapeutics, discusses gene therapy being developed to treat children with metachromatic leukodystrophy (MLD). MLD is a lysosomal storage disorder characterized by the accumulation of sulfatides in cells....
by Peter Ciszewski | Nov 22, 2019
Rajiv Ratan, MD, PhD, of the Burke Neurological Institute, explains hemorrhagic stroke. A hemorrhagic stroke occurs when a blood vessel bursts and bloof leaks into the surrounding brain tissue. Surprisingly, this type of stroke is relatively rare (compared to...
