Professional Track
Kimberly A Chapman, MD, PhD
"Rare-X for Research and Medical"
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Dr. Ferdinand von Meyenn
"Establishing MTHFR- and MTR- deficient Cerebral Organoids"
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Dr. Brian Gilfix, MDCM, PhD, FRCPC DABCC, FADLM
"Aminoglycosides for the Treatment of Vitamin B12 Metabolism (cblG) l"
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Ken Maclean, PhD
"Combining Taurine and Formate/Betaine Treatment to Improve Clinical Outcomes and Drug Safety in Classical CBS Deficient HCU"
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Eric Dube, PhD.
"Harmony Study, Phase 3 Clinical Trial of Pegitbatinase - Travere"
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Matt Lanchantin
"Investigating the Therapeutic Potential of Intestinal Methionine Elimination via Oral Enzyme Therapy in HCU - Syntis Bio"
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Family Track
Janet A Thomas, MD
"What is Homocystinuria?"
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Kimberly A Chapman, MD, PhD
"Treatment Guidelines"
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Eirini Manoli, MD, PhD
"Treatment Guidelines"
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Warren Kruger, PhD
"What is: ERT, Chaperone Therapy, Gene Editing, Gene Therapy"
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Brittany Parke
"Classical HCU and Remethylation Research Overview"
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Kristen
"Research: What you should know and ways to get involved"
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