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