Rare and Genetic Disease Network
Jim Schulz, Co-founder and Chief Financial Officer of Scioto Biosciences, discusses his company’s focus on developing therapies devoted to having a impact on the delivery of microbiome therapeutics with the goal of reducing the incidence of necrotizing enterocolitis (NEC).
Gail Cawkwell, MD, PhD, Senior Vice President, Medical Affairs at Intercept, provides an overview of primary biliary cholangitis (PBC), a rare liver disease that is caused by an autoimmune reaction.
Richard West, Founder and CEO of Baebies, a newborn testing diagnostic company, provides an overview of his platform.
Menkes disease is a disorder that affects copper levels in the body. It is characterized by sparse, kinky hair; failure to thrive; and progressive deterioration of the nervous system.
Brian Schwartz, Chief Medical Officer of ArQule, discusses his company and it’s clinical-stage pipeline consistlng of five drug candidates, all of which are in targeted, biomarker-defined patient populations.
Richard West, Founder and CEO of Baebies, a newborn testing diagnostic company.
Heather A. Lau, MD, Director, Lysosomal Storage Disease Program at NYU Langone in New York City discusses Mucopolysaccharidosis type III (MPS III), also known as Sanfilippo syndrome.
Dr. Shenda Baker is Chief Executive Officer of Synspira, a clinical stage biotech company developing a new class of glycopolymer-based therapeutics that have the potential to address a major unmet need associated with pulmonary disorders-
Alnylam Pharmaceuticals announced the publication of the pivotal study results from the APOLLO Phase 3 trial of patisiran in The New England Journal of Medicine (NEJM).
Ron Cooper, President and CEO of Albireo, provides and overview of progressive familial intrahepatic cholestasis (PFIC), a rare genetic condition that affects the liver. PFIC is estimated to affect between one in every 50,000 to 100,000 children born worldwide and causes progressive, life-threatening liver disease.
James McArthur, PhD, Founder of Imara and President, R&D of Cydan discusses the development of IMR-687 for the treatment or rare hemoglobinopathies.
Manu Nair, Vice President of Technology Ventures at Oklahoma Medical Research Foundation (OMRF) discusses his group’s focus on building partnerships with industry that advance the research in OMRF’s laboratories.