by Peter Ciszewski | Aug 23, 2018
Heather A. Lau, MD, Director, Lysosomal Storage Disease Program at NYU Langone in New York City, discusses her experience working with rare diseases and neurogenetics.
by Peter Ciszewski | Aug 23, 2018
The U.S. Food and Drug Administration approved cenegermin (Oxervate), for the treatment of neurotrophic keratitis, a rare disease affecting the cornea (the clear layer that covers the colored portion of the front of the eye). This is the first approved drug for this...
by Peter Ciszewski | Aug 21, 2018
Editor’s Note: This interview was conducted at #Bio2018. Please pardon the background noise. Brian Schwartz, Chief Medical Officer of ArQule, discusses his company and it’s focus on disease management. ArQule is engaged in the research and...
by Peter Ciszewski | Aug 17, 2018
Luca Santarelli, MD of Therachon discusses his company’s lead pipeline candidate, TA-46, a novel protein therapy in development for achondroplasia, the most common form of short-limbed dwarfism. Achondroplasia is an autosomal dominant disorder...
by Peter Ciszewski | Aug 16, 2018
The U.S. Food and Drug Administration (FDA) has approved ivacaftor (KALYDECO) to be used in children with cystic fibrosis ages under the age of 2 years old who have at least 1 mutation in their cystic fibrosis transmembrane conductance regulator (CFTR) gene that is...
