Day of Hope Conference Presenter Abstracts
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Cystinosis: The Building Blocks
Stephen Jenkins, MD, CRF Trustee
University of Utah Hospital
Cystinosis, The Early Years: Fanconi Syndrome and More
Larry Greenbaum, MD, PhD
Emory University School of Medicine and Children’s Healthcare
The Changing Face of Cystinosis from Childhood to Adulthood
Julian Midgley, BM, BCh
Alberta Children’s Hospital
Advances in Kidney Care and Transplantation
Paul Grimm, MD
Stanford University/Lucile Packard Children’s Hospital
Regenerating Kidney Tissue to Combat Nephropathic Cystinosis
Benjamin Freedman, PhD
University of Washington
Current Ocular Therapy for Cystinosis
Donny Suh, MD, FAAP, MBA, FACS
Gavin Herbert Eye Institute at University of California, Irvine
Development of a Controlled Release Cysteamine Eye Drop for Corneal Cystinosis
Morgan DiLeo, PhD
University of Pittsburgh, School of Medicine
CCIR: Be Part of the Cure
Clay Emerson, PhD, PE, CRF Trustee
Translational Approaches for Cystinosis, New Targets, Same Hope
Sergio Catz, PhD
The Scripps Research Institute
Preclinical Studies on New Treatments for Cystinosis in Cystinotic Rats
Jennifer Hollywood, PhD
University of Auckland, New Zealand
Why Patient Voices Matter: Cystinosis & Revolutionizing Gene Therapies for Rare Diseases and Beyond
Betty Cabrera, MPH
University of California, San Diego
Phase 1/2 Clinical Trial of Autologous Hematopoietic Stem and Progenitor Cell Gene Therapy for Cystinosis
Stéphanie Cherqui, PhD
University of California, San Diego
Illuminating Novel Molecular Mechanisms of Lysosomes in Cystinosis to Uncover Therapeutic Targets
Lauren Albrecht, PhD
University of California, Irvine
Intracellular Cystine Measurement. What is Normal?
Bruce Barshop, MD, PhD
University of California, San Diego
An mRNA/Lipid-Nanoparticle Strategy to Treat Cystinosis
Paul Goodyer, MD
McGill University Health Center in Montreal, Canada