Professional Track
Dr. Ferdinand von Meyenn
"Establishing MTHFR- and MTR- deficient Cerebral Organoids"
Dr. Brian Gilfix, MDCM, PhD, FRCPC DABCC, FADLM
"Aminoglycosides for the Treatment of Vitamin B12 Metabolism (cblG) l"
Ken Maclean, PhD
"Combining Taurine and Formate/Betaine Treatment to Improve Clinical Outcomes and Drug Safety in Classical CBS Deficient HCU"
Eric Dube, PhD.
"Harmony Study, Phase 3 Clinical Trial of Pegitbatinase - Travere"
Click here to read his bio
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
"Research: What you should know and ways to get involved"
Click here to read her bio
