The HCU Network America 3rd patient-expert conference was held June 25-26, 202022, in Bethesda, MD. Over 150 patients, parents, relatives, medical professionals, researchers, and industry reps from 26 states and 3 additional countries came together to celebrate our Brave HCU Heroes!
Conference Program
Conference Report
Poster Presentations
Not included on site:
Travere: Mehul Shah, MD, PhD
Pegtibatinase, an Investigational Enzyme Replacement Therapy for the Treatment of Classical Homocystinuria: Initial Results from the Phase 1/2 COMPOSE Study
NIH: Pauline Hoffman
A translational approach to study combined methylmalonic acidemia and homocysteinemia cblC type.
NIH: Abigael Gebremariam
Exploring the natural history of cobalamin B (cblB) type methylmalonic acidemia (MMA) to guide the development of systemic AAV gene therapy.
