by Peter Ciszewski | Nov 19, 2018
Daniel Glaze, Medical Director of the Blue Bird Circle Rett Center and a Professor in the Departs of Pediatrics and Neurology at Baylor College of Medicine in Houston discusses the Rett Syndrome Natural History Study. The goal of the Natural History Study is to...
by Peter Ciszewski | Nov 18, 2018
Rachelle Dixon, President of the HSAN1E Society discusses the advocacy group she co-founded for hereditary sensory and autonomic neuropathy type 1E (HSAN1E), a progressive disorder of the central and peripheral nervous systems. Symptoms of this rare disease...
by Peter Ciszewski | Nov 17, 2018
Barry Ticho, MD, Chief Medical Officer at Stoke Therapeutics, provides an overview the company’s TANGO (Targeted Augmentation of Nuclear Gene Output) technology. TANGO allows for precisely modulating splicing to increase a target protein’s expression by...
by Peter Ciszewski | Nov 16, 2018
Richard A. Basile Co Founder and CEO of BioPontis Alliance for Rare Diseases, discusses his organization’s focus on rare diseases, with two programs on Charcot-Marie-Tooth disease and Huntington’s Disease. BioPontis is an international nonprofit...
by Peter Ciszewski | Nov 15, 2018
Katherine Stueland, Chief Commercial Officer at Invitae, provides an overview of her company and their attendance at this year’s 8th Orphan Drugs and Rare Diseases conference in Boston, MA in September. Invitae is a genetic information company bringing...
