Patient Volunteers

The Cystinosis Research Foundation is dedicated to funding research that will lead to improved treatments and a cure for cystinosis. The following studies are being funded by the CRF to advance knowledge in key areas of cystinosis treatment.  Click the following links to see the full information for each study.

Phase 3 Trial Planned for Delayed Release Cysteamine, Raptor Pharmaceuticals - 2nd Location Announced!

We are proud to announce that Dr. Ranjan Dohil's research studies, funded by CRF, resulted in the development of a delayed-release form of cysteamine.  Raptor Pharmaceuticals Corp. acquired the worldwide license for the delayed-release form of cysteamine from UCSD.  They are committed to advancing the slow-release drug and have filed an investigational Drug Application with the FDA.  Phase 3 Trials are now underway at Emory University, Atlanta, Georgia and Children's Memorial Hospital in Chicago, Illinois. Additional locations will be announced in the very near future, what for further information.

"Brain and Cognitive Development in Cystinosis," UCSD Pediatric Neurology Research Group

Individuals will be asked to perform such tasks as putting together puzzles, defining words, and identifying or remembering pictures. The testing is non-invasive and behaviorally oriented, and it helps us learn more about the cognitive and behavioral effects of cystinosis.

"Executive Functioning in Cystinosis,"  UCSD Pediatric Neurology Research Group

This study will examine problem solving, planning, attention, and impulsivity. The testing is non-invasive and behaviorally-oriented, and it will help us learn more about executive functioning in cystinosis. Individuals will also be asked to fill out a questionnaire regarding behaviors such as planning and attention.

"Stress and Coping in Cystinosis," UCSD Pediatric Neurology Research Group 

This study will examine stress reactions, coping responses, quality of life, and medical adherence in individuals with cystinosis.  Individuals with cystinosis ages 12 years through adulthood and their parents will be asked to complete several questionnaires.