News

Giving Tuesday Exceeded Our Goal Thank You!

$206,705 Raised for Cystinosis Research Your generosity on Giving Tuesday was astounding.  Together we raised more than $206,705 for cystinosis research! As a result of your commitment to CRF’s research program, CRF has funded 212 research studies in 12 countries ensuring that there is a CRF researcher working on cystinosis every minute of the day. CRF’s highly …

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Research Grant Progress Report by Justine Bacchetta, MD, PhD, Hospices Civils de Lyon, France

CRF awarded Justine Bacchetta, MD, PhD, and Irma Machuca-Gayet, PhD, their first cystinosis research grant in 2018, for the study of cysteamine toxicity on the bone which became known as the CYSTEABONE project. The second grant was awarded in the fall of 2020, to further study the pathophysiology of bone disease in nephropathic cystinosis.  This …

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Research Progress Report by Morgan Fedorchak, PhD, and Ken Nischal, MD, FRCO, University of Pittsburgh

Morgan Fedorchak, PhD, Assistant Professor, and Ken Nischal, MD, FRCO, Professor, Chair of Pediatric Ophthalmology at the University of Pittsburgh, received their most recent grant award in spring 2020 for their work to develop a controlled release cysteamine eye drop. CRF has supported and funded their important research for corneal cystinosis since 2016 with $880,258 …

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Research Grant Progress Report by Liang Feng, PhD, Stanford University

Researcher Liang Feng, Associate Professor of Molecular and Cellular Physiology at Stanford University, and Research Fellow Xue Guo received a grant award in 2020 to continue their work on the Molecular Mechanism of Cystinosis. The objectives of this study are to understand several key aspects of how membrane proteins that have important implications in cystinosis …

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Final Report – Development and Characterization of a Rat Model of Cystinosis

In 2018, CRF awarded a grant to Olivier Devuyst, MD, PhD, University of Zurich, Switzerland, and Francesco Emma, MD, Bambino Gesù Pediatric Hospital, Rome, Italy, for their research on the Development and Characterization of a Rat Model of Cystinosis.  They successfully generated a novel Ctns-/- rat model that replicates the clinical symptoms of human nephropathic …

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Research Progress Report by Jennifer Hollywood, PhD, and Alan Davidson, PhD, University of Auckland, New Zealand

The goal of this study is to conduct therapeutic drug intervention studies in Ctns knock-out rats to determine whether a combination treatment of cysteamine and Everolimus provides greater therapeutic benefit than cysteamine alone. CRF has granted research funding to the University of Auckland since 2009, awarding $805,143 for the important research conducted by Dr. Hollywood …

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Research Progress Report by Michael Sekar, PhD, AMMA Therapeutics, Inc.

In the spring of 2020, CRF awarded a grant to AMMA Therapeutics for $304,064, the aim of their study is to develop a once-daily subcutaneous injection of cysteamine bitartate.  The project is led by Michael Sekar, PhD, Principal Investigator, and Laura Rita Rega, PhD, Co-Principal Investigator. The project is comprised of three specific Aims.  Aim …

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