by Peter Ciszewski | Sep 9, 2018
David Birch, PhD, Scientific Director at the Retina Foundation discusses X-linked retinoschisis (XLRS) is an inherited early onset retinal degenerative disease caused by mutations in the RS1 gene. It is the leading cause of juvenile macular degeneration in males. XLRS...
by Peter Ciszewski | Sep 8, 2018
Amit Rakhit, MD, Chief Medical and Portfolio Officer at Ovid Therapeutics, provides an overview on Angelman syndrome. Angelman syndrome is a complex genetic disorder that primarily affects the nervous system. Characteristic features of this condition include delayed...
by Peter Ciszewski | Sep 7, 2018
The US Food and Drug Administration (FDA) approved Tiglutik (riluzole) oral suspension for the treatment of amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease. The product was previously granted Fast Track Designation and Orphan Drug...
by Peter Ciszewski | Sep 6, 2018
U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for ATI-1013, a fully human, anti-nicotine monoclonal antibody for the treatment of Buerger’s Disease. Buerger’s Disease (thromboangitis obliterans) is a rare disorder that is caused by...
by Peter Ciszewski | Sep 5, 2018
Heather A. Lau, MD, Director, Lysosomal Storage Disease Program at NYU Langone in New York City discusses Pompe disease, an inherited disorder caused by the buildup of a complex sugar called glycogen in the body’s cells. The accumulation of glycogen in...
