Join Denny Sanford and Dr. David Brenner to celebrate a day of unity, breakthrough scientific discoveries, and therapies for children’s disorders.
This event will be In-person only. There will not be a hybrid/zoom option available for attendees.
Program
| Welcome
T. Denny Sanford Namesake and Honorary Trustee |
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| Session Chair
David A. Brenner, MD President and CEO, Sanford Burnham Prebys |
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| Sanford Children’s Health Research Center Faculty | |
| José Luis Millán, PhD | “Developing therapeutics for soft bones and ectopic calcification disorders†|
| Duc Dong, PhD | “From hope for few to drug for many – Why rare is precious†|
| Evan Snyder, MD PhD | “A clinical trial using human neural stem cells for neuroprotection in perinatal asphyxia, a major cause of cerebral palsy in kids†|
| Anne Bang, PhD | “Drug screens of human induced pluripotent stem cell (hiPSC) derived neuronal networks on multi-electrode arrays” |
| Pamela Itkin-Ansari, PhD | “Proinsulin misfolding in diabetes†|
| Yu Yamaguchi, MD PhD | “Multiple hereditary exostoses – From genetics to potential drug targets” |
| Hudson Freeze, PhD | “Fucose therapy: Revising bedrock biochemistry” |
| Lunch | |
| Session Chair
Hudson Freeze, PhD Professor and Director, Sanford Children’s Health Research Center |
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| Sanford Health Sioux Falls, SD | |
| David Pearce, PhD | “From rare diseases to type-1 diabetes: Research that impacts children at Sanford Health†|
| Frontiers in Congenital Disorders of Glycosylation Consortium | |
| Eva Morava, MD, PhD | “Clinical trials in Glyco-land” |
| Ethan Perlstein, PhD | “Precision drug repurposing: patient avatar to pioneer study to Phase 3 trial” |
| UC San Diego | |
| Lars Bode, PhD | “Human milk-based therapeutics and diagnostics to protect preterm babies from necrotizing enterocolitis†|
| Stephanie Cherqui, PhD | “Hematopoietic stem cell gene therapy for cystinosis: Mechanism of action and clinical trial update” |
| Reception | |
| T. Denny Sanford | Welcome patients and families
Speakers introduce their guest patients and families. |
Professor
Human Genetics Program
Sanford Children’s Health Research Center
Professor
Human Genetics Program
Sanford Children’s Health Research Center
Associate Dean of Admissions
Graduate School of Biomedical Sciences
Associate Professor
Human Genetics Program
Sanford Children’s Health Research Center
Professor
Human Genetics Program
Sanford Children’s Health Research Center
Director, Center for Stem Cells and Regenerative Medicine
Director
Cell Biology
Conrad Prebys Center for Chemical Genomics
Adjunct Associate Professor
Development, Aging and Regeneration Program
Center for Genetic Disorders and Aging Research
Director
Sanford Children’s Health Research Center
Director and Professor
Human Genetics Program
Sanford Children’s Health Research Center
President
Innovation, Research, and World Clinic
Sanford Health
Director
Frontiers in Congenital Disorders of Glycosylation Consortium
Consultant, Department of Clinical Genomics
Mayo Clinic
Founder and CEO
Perlara, Inc.
Professor of Pediatrics
Endowed Chair of Collaborative Human Milk Research
UC San Diego
Professor of Pediatrics
UC San Diego