2018 NPKUA CONFERENCE
GENERAL SESSIONS

2018 NPKUA Conference Breakout Sessions
2018 NPKUA Conference Scientific Poster Session

Friday, July 6, 2018

Welcome and NPKUA Update
Amy J. Oliver, JD, President and Christine S. Brown, MS, Executive Director
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The Discovery of PKU

Geir Ivar Følling Elgio
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The Future of PKU Drug Development
Betsy Bogard
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International Association Introduction
Tobias Hagedorn, Secretary of E.S.PKU
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PKU Patient Registry Update
Eileen Blakely, MS. RD, CDN, Registry Coordinator
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The Discovery of PKU
Geir Ivar Følling Elgio
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NPKUA FUNDED RESEARCH PANEL
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One of the biggest accomplishments of the NPKUA is the creation of a research grant program to drive more research for PKU. This panel highlights the critical and innovative work of some of our current funded projects.

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Humanization of a Genetically Modified Probiotic to Treat PKU
Dr. Katherine Durrer, Post-Doctoral Research Fellow at University of North Texas Health Science Center
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Characterization of Immunomodulatory Properties in human Amnion Epithelial Cells for Cellular Therapy
Dr. Roberto Gramignoli, Karolinska Instituet, Stockholdm Sweden
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A New Approach to Pharmacologic Chaperones for PKU Therapy
Dr. Eileen Jaffe, Fox Chase Center
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Neurobehavorial Features of a Pig Model of PKU
Dr. Robert Nicholls, Professor of Pediatrics in the Division of Medical Genetics at the Children's Hospital of Pittsburgh of UPMC
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CRISPR-Cas9 Mediating Gene Correction in the PKU Mouse Model
Dr. Cary Harding, Professor of Molecular and Medical Genetics and Pediatrics and Daelyn Richards, Doctoral Candidate, Oregon Health & Science University
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Development of Gene Editing Systems to Treat PKU
Dr. Yizhou Dong, Assistant Professor at Ohio State University
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Home Phe Monitor Update
Dr. Tom Franklin
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Adult and Teen Panel Discussion
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This engaging panel looks at ways drug developers are currently looking at various therapeutic approaches to treat, and even cure, PKU. The panel also includes information of the recent approval of Palynziq to treat PKU Adults.

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Emerging Therapies Panel Discussion
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Palynziq
Presenting: Dr. Cary Harding, MD, FACMG, Professor of Molecular and Medical Genetics and Pediatrics, Oregon Health & Sciences University
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A Novel Class of Living Medicines
Aoife Brennan, MB, BCh, BAO, MMSc, Cheif Medical Officer, Synlogic, Inc.
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CDX-6114: an orally administered enzyme for the potential treatment of PKU
Gjalt, Huisman, PhD, Vice President of Biotherapeutics, Codexis, Inc.
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Hepatocyte Transplantation for Liver Based Metabolic Disease
Presenting: Dr. Robert Nicholls, Professor of Pediatrics, Division of Medical Genetics at the Children's Hospital of Pittsburgh of UPMC
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Red Cells and PKU
Dr. Chris Charpenter, Chief Medical Officer, Rubius
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Clinical Trials Panel Discussion
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This interactive panel discusses the clinical trial process from the perspective of the FDA, a pharmaceutical company, a principal investigator and two former clinical trial participants.

 

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