Visionary Award
Recipient
Margie McGlynn
HCU Hero Award
Recipient Keynote
Professional Track
Dr. Ferdinand von Meyenn
"Establishing MTHFR- and MTR- deficient Cerebral Organoids"
Beata Vekeriotaite
"Establishing MTHFR- and MTR- deficient Cerebral Organoids"
Dr. Brian Gilfix, MDCM, PhD, FRCPC DABCC, FADLM
"Aminoglycosides for the Treatment of Inborn Errors of Vitamin B12 Metabolism (cblG)"
Ken Maclean, PhD
"Combining Taurine and Formate/Betaine Treatment to Improve Clinical Outcomes and Drug Safety in Classical CBS Deficient HCU"
Julie Lin, MD, MPH, FASN
"Harmony Study, Phase 3 Clinical Trial of Pegitbatinase - Travere"
Ashley Hudson
"Investigating the Therapeutic Potential of Intestinal Methionine Elimination via Oral Enzyme Therapy in HCU - Syntis Bio"
Family Track
Warren Kruger, PhD
"What is: ERT, Chaperone Therapy, Gene Editing, Gene Therapy"
Kristen Skvorak, PhD
"Research: What you should know and ways to get involved"
