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Studies of Inflammation in Cystinosis
Sergio Catz, PhD
Scripps Research

Phase I and II Clinical Trial of Autologous Hematopoietic Stem and Progenitor Cell Gene Therapy for Cystinosis
Stéphanie Cherqui, PhD
University of California, San Diego

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

The Search for Additional Treatments in Nephropathic Cystinosis
Francesco Emma, MD
Bambino Gesù Children’s Hospital

Lessons from the European Patient Population
Francesco Emma, MD
Bambino Gesù Children’s Hospital

Executive Function in Cystinosis
Ana A Francisco, PhD and Sophie Molholm, PhD
Albert Einstein School of Medicine

Changing Urine Into Kidney to Combat Cystinosis
Benjamin Freedman, PhD
University of Washington

Cystinosis Medication Management Throughout the Life Course
Paul Grimm, MD
Stanford University/ Lucile Packard Children’s Hospital

Planning (long-term and short-term) for Kidney Transplant and Living Your Best Life Possible After Kidney Transplant
Paul Grimm, MD
Stanford University/ Lucile Packard Children’s Hospital

Cystinosis: The Building Blocks
Stephen Jenkins, MD, CRF Trustee
University of Utah Hospital

A Cohort of 23 Individuals with Cystinosis: Up to 30 Years of Follow-Up
Julian Midgley, BM, BCh
Alberta Children’s Hospital

Muscle Involvement and Swallowing Assessments
Reza Seyedsadjadi, MD
Massachusetts General Hospital