Sep 17, 2020
Raj Kannan, CEO, Chiasma is focused on bringing to market oral formulations to patients that face significant challenges with their existing injectable therapies. Targeting those suffering from the rare disease acromegaly, recent approval of MYCAPSSA brings the first and only oral somatostatin analog to a predominantly...
Sep 17, 2020
Raj Kannan, CEO, Chiasma is focused on bringing to market oral formulations to patients that face significant challenges with their existing injectable therapies. Targeting those suffering from the rare disease acromegaly, recent approval of MYCAPSSA brings the first and only oral somatostatin analog to a predominantly...
Addressing Genetic Mutation that Leads to Rare Disease of the Immune System with Paula Ragan X4 Pharmaceuticals TRANSCRIPT
Download PDFAug 25, 2020
Paula Ragan, CEO and President, X4 Pharmamceuticals is developing innovative approaches to treat patients suffering from rare diseases with an initial focus on WHIM syndrome, a rare disease of the immune system that has a basis in a genetic mutation. X4 is also focused on a rare form of lymphoma called Waldenstrom...
Aug 25, 2020
Paula Ragan, CEO and President, X4 Pharmaceuticals is developing innovative approaches to treat patients suffering from rare diseases with an initial focus on WHIM syndrome, a rare disease of the immune system that has a basis in a genetic mutation. X4 is also focused on a rare form of lymphoma called Waldenstrom...
Gene Silencing Driving Drug Development for Rare Diseases with Dietrich Stephan NeuBase Therapeutics TRANSCRIPT
Download PDFAug 12, 2020
Dietrich Stephan PhD, CEO, NeuBase Therapeutics discusses the history of research into the genetic basis of all diseases, NeuBase's PATrOL™ platform allowing for intelligent design of drugs that engage with a malfunctioning gene, and the company's therapies in development for Huntington's disease and myotonic...