Rare and Genetic Disease Network


Scioto Biosciences: Focused on Reducing Necrotizing Enterocolitis

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).

Menkes Disease

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.

Sanfilippo Syndrome (MPS III) Overview

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.

Synspira: Focusing on Rare Pulmonary Diseases

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-

Progressive Familial Intrahepatic Cholestasis (PFIC) Overview

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.

Special Reports