HCU Network America's 4th patient-expert conference was held June 29-30, 2024, in Aurora, CO. Approximately 200 patients, parents, relatives, medical professionals, researchers, and industry reps from 29 states and 2 additional countries came together to help participate in Moving Mountains for HCU!
Conference Program
Diagnostics, Monitoring, and Natural History
Classical Homocystinuria Research
Remethylation Disorders Research
Ask the Expert
Poster Presentations
Not included on site:
Kimberly Champman, PhD, Children’s National
HCU Network America Surveys help define what is important to patients
Kimberly Champman, PhD, Children’s National
When to look for homocystinuria: Preliminary results from HCU Network
America’s Patient surveys.
Irini Manoli, MD, NIH
Hydroxocobalamin Dose Intensification Improves Ocular, Neurological, and
Biochemical Outcomes in CblC Deficiency
Joseph Schinaman, PhD, Petri Bio
A microbiome-mediated dietary treatment for classical homocystinuria
Jalé Güner, Travere Therapeutics
Latest Results From the COMPOSE® Phase 1/2 Trial for the Treatment of Clas-sical Homocystinuria (HCU) Using Pegtibatinase, a Novel Investigational En-zyme Replacement Therapy
