Day of Hope Conference Presenter Abstracts
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Repurposing Drug Libraries and Artificial Intelligence for the Discovery of New Treatments for Cystinosis
Sergio Catz, PhD
The Scripps Research Institute, La Jolla, CA
Hematopoietic Stem Cell Gene Therapy for Cystinosis: Updated Results From the Phase 1/2 Clinical Trial
Stéphanie Cherqui, PhD, CRF Trustee
University of California, San Diego
Ocular Biodistribution of Cysteamine Delivered by a Sustained Release Microsphere/ Thermoresponsive Gel Eye Drop
Morgan V. DiLeo, PhD
University of Pittsburgh – School of Medicine
CCIR and You – Help Us Find A Cure
Clay Emerson, PhD, PE, CFM
Using Human Pluripotent Stem Cells to Develop New Treatments For Cystinosis
Benjamin S. Freedman, PhD
University of Washington
Kidney Transplant Smorgasbord
Paul C. Grimm, MD
Stanford University School of Medicine
Managing All Those Supplements When Your Child or You Have Fanconi Syndrome
Paul C. Grimm, MD
Stanford University School of Medicine
Cystinosis: Back to the Basics
Stephen Jenkins, MD, CRF Trustee
University of Utah Hospital
Advancing the Understanding of the Renal Fanconi Syndrome in Cystinosis
Veenita Khare, PhD
University of California, San Diego
Muscle Wasting in Children with Cystinosis
Robert Mak, MD, PhD
Rady Children’s Hospital
University of California, San Diego
The Varied Challenges of Cystinosis for Young Adults and Adults
Julian Midgley, BM BCh
Alberta Children’s Hospital, Calgary, Alberta, Canada
Myopathy and Dysphagia in Adults with Cystinosis
Stacey Sullivan, MS, CCC-SLP, and Natalie Grant, BA
Massachusetts General Hospital