by Peter Ciszewski | Nov 10, 2018
Daniel Glaze, Medical Director of the Blue Bird Circle Rett Center and a Professor in the Departments of Pediatrics and Neurology at Baylor College of Medicine in Houston, discusses ongoing Rett syndrome research and clinical trials, including the ongoing phase...
by Peter Ciszewski | Nov 8, 2018
Rachelle Dixon, President and co-founder’s of the HSAN1E Society discusses hereditary sensory and autonomic neuropathy type 1E (HSAN1E), a progressive disorder of the central and peripheral nervous systems. Symptoms of this rare disease typically begin by age...
by Peter Ciszewski | Nov 6, 2018
Barry Ticho is the Chief Medical Officer at Stoke Therapeutics, and in this clip he provides an overview of his company. Stoke Therapeutics is a biotechnology company working to increase gene expression to treat a wide array of severe genetic diseases,...
by Peter Ciszewski | Nov 5, 2018
The Food and Drug Administration (FDA) approved an oral film formulation of clobazam (Sympazan, Aquestive Therapeutics) for the adjunctive treatment of seizures associated with Lennox-Gastaut syndrome (LGS) in patients 2 years and older. Sympazan is the first oral...
by Peter Ciszewski | Nov 5, 2018
Richard A. Basile,Co-Founder and CEO of BioPontis Alliance for Rare Diseases, discusses the challenges of developing drugs for rare diseases. BioPontis is an international nonprofit organization whose mission is to advance promising science into potential...
