About Us

History

Cystinosis research has expanded tremendously in recent years.  Cystinosis researchers and scientists have made significant research progress and their results are increasingly translated to clinical trials.  In light of the ongoing cystinosis research projects around the world and the anticipation of new, clinical trials on the horizon, the Cystinosis Research Foundation recognized the need to create a hub of information for researchers and scientists who are pursuing better treatments and a cure for cystinosis.

In 2009, leaders in the cystinosis community began discussions on the establishment of a new kind of resource that would connect and serve the needs of the entire cystinosis community.  In order to further the research effort and to accelerate the research process, the Cystinosis Research Foundation (CRF) in collaboration with the Cystinosis Foundation (CF) and other key cystinosis leaders and organizations around the world, worked to create a patient based international cystinosis registry. What we envisioned was the creation of a central hub for information on cystinosis that would bring together those living with cystinosis, their families, friends and caregivers and connect them with the medical research community, clinical care and clinical trials.  The information provided by the patients and families would then be made available to clinicians, scientists, researchers and other stakeholders in the cystinosis community in order to discover novel treatments and a cure for cystinosis.

This hub of information available as a resource for industries with an interest in cystinosis, will provide them limited access to a database of information provided by patients and their families – information that could prove vital to advances in the care and treatment for those with cystinosis. Today, the result of this endeavour is the Cure Cystinosis International Registry (CCIR), a cutting-edge web site and international registry.

For patients, CCIR will provide an opportunity for involvement in research that will help develop and test new therapies and develop a cure for cystinosis.  The registry resource will connect the stakeholders in the cystinosis community – the scientists, researchers, clinicians, pharmaceutical companies, patients and families - and provide them with resources that have never been available in one place before. The CCIR is dedicated to improving the lives of those affected by cystinosis and to finding a cure for cystinosis. 

Who We Are

CCIR is the only comprehensive, global registry for cystinosis.  The purpose and goal of CCIR is to achieve worldwide registration of the cystinosis community  which will increase the community’s ability to help one another through improved communication and information sharing and by doing so, help the thousands of people affected by cystinosis.  Among other things, CCIR is a central hub for current information regarding cystinosis clinical trials and studies. 

The Cure Cystinosis International Registry represents the collaboration of a wide range of entities and individuals who recognize the value and importance of working together, combining their expertise and information.  Regardless of their individual backgrounds, the thought leaders of CCIR share a common goal - to increase the community’s ability to help one another through improved communication and cooperation and by doing so help those affected by cystinosis. The organizers of CCIR come from leaders in the non-profit sector, academic institutions and the scientific community. This group of people are focused on their desire to accelerate the research process in their quest to find the cure for cystinosis.

The information provided on the CCIR has been developed with the review, direction and advice from the CCIR Medical and Scientific Council, a group of experts in cystinosis, and the CCIR Advisory Board. The CCIR Advisory Board contributes regularly to the CCIR in many areas including reviewing educational materials, providing programmatic direction, advising on the processes and procedures of the registry and facilitating opportunities for outreach and collaboration. The CCIR Advisory Board is dedicated to promoting and facilitating current research and medical information to the global cystinosis community in an effort to inform the community of current treatments and clinical trials.  The CCIR Advisory Board’s purpose is to ensure that information gathered through CCIR will be available to the cystinosis research community to evaluate therapies, to design clinical trials for improved therapies, including a cure for cystinosis and to further understand cystinosis and its complications.  The Operations Team behind this incredible resource continues to refine and perfect the site so that it evolves to best serve the needs of the entire cystinosis community. We encourage your suggestions and feedback.

CCIR Partners

Cystinosis Research Foundation, www.cystinosisresearch.org
Cystinosis Foundation, www.cystinosisfoundation.org

CCIR Operations Team

Bruce A. Barshop, MD, PhD, University of California, San Diego
Kyle Brown, CEO, Innolyst, Inc
Betty L. Cabrera, M.P.H., University of California, San Diego
Jason Grier, Hope for Holt
Jan Laibe, Innolyst, Inc.
Nancy Stack, Cystinosis Research Foundation

CCIR Board of Advisors

Bruce A. Barshop, MD, PhD, University of California, San Diego
Kyle Brown, CEO, Innolyst, Inc
Betty L. Cabrera, M.P.H, University of California, San Diego
Paul Goodyer, MD, Montreal Children's Hospital, Montreal, Canada
Whitney Glaize, Cystinosis Patient Advocate, Cystinosis Research Foundation
Jean Hobbs-Hotz, President, Cystinosis Foundation
Valerie Hotz, Cystinosis Foundation
Tom Melang, Cystinosis Patient Advocate
Patrick Niaudet, MD, Hôpital Necker-Enfants Malades, Paris, France
Teresa Partington,  Jenna and Patrick's Foundation of Hope
Jerry Schneider, MD, Dean for Academic Affairs Emeritus, University of California, San Diego
Nancy Stack, Cystinosis Research Foundation
Jody Strauss, Cystinosis Awareness and Research Effort 
Tricia Sturgis,  24 Hours for Hank
Doris A.Trauner, MD, University of California, San Diego

CCIR Medical and Scientific Council

Bruce A. Barshop, MD, PhD, University of California, San Diego
Stephanie Cherqui, PhD, The Scripps Research Institute
Ranjan Dohil, MD, University of California, San Diego
Paul Goodyer, MD, Montreal Children's Hospital, Montreal, Canada
Daniel R. Salomon, MD, The Scripps Research Institute
Jerry Schneider, MD, Dean for Academic Affairs Emeritus, University of California, San Diego
Doris A.Trauner, MD, University of California, San Diego

CCIR Advocates

24 Hours for Hank, www.24hoursforhank.org
Australian Cystinosis Support Group, www.cystinosis.com.au
CARE, Cystinosis Awareness and Research Effort, Canada, www.cystinosis.ca
Cystinosis Foundation New Jersey Chapter
Cystinose Foundation France, www.cystinose.org
Cystinosis Foundation Ireland, www.cystinosis.ie
Cystinosis Foundation UK, www.cystinosis.org.uk
Cystinosis Support Group, South Africa
Hope for Holt, www.hopeforholt.org
Jenna and Patrick's Foundation of Hope, www.jennaandpatrick.org
Joshua's Journey of Hope, www.joshuasjourney.org
Tina's Hope for a Cure, www.tinashopeforacure.org