The Genetic Disorders Mucociliary Clearance Consortium

Stephanie Davis, MD, and Thomas Ferkol, MD,  Professors of Medicine at University of North Carolina at Chapel Hill, discuss the value of being part of the Rare Diseases Clinical Research Network (RDCRN). Drs. Davis and Ferkol are co-leaders of the Genetic Disorders...

Panel Discussion: The High Cost of Rare Diseases  

Joni Rutter, PhD, Acting Director at the National Center for Advancing Translational Sciences (NCATS) and Annie Kennedy, Chief of Policy and Advocacy at the EveryLife Foundation discuss their organizations’ collective studies showing the direct and indirect...

The United MSD Foundation

Sarah Cortell Vandersypen, Executive Director of the United MSD Foundation, discusses the organization’s work with multiple sulfatase deficiency.     The United MSD Foundation is an international patient advocacy group focused on providing support for...