News

Morgan DiLeo, 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 …

Research Progress Update by Morgan DiLeo, PhD, University of Pittsburgh Read More »

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 …

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

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 …

Research Grant Progress Report by Liang Feng, PhD, Stanford University Read More »

Stem Cell and Gene Therapy Trial Update with Dr. Stéphanie Cherqui, Jordan Janz, and Barb Kulyk Presented Tuesday, October 27, 2020 We had a record of 309 people participating from 20 countries around the world.  The participants represented the myriad of people in our community; adults with cystinosis, parents of children and adults with cystinosis, …

Stem Cell And Gene Therapy Webinar Update Video Now Available! Read More »

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 …

Final Report – Development and Characterization of a Rat Model of Cystinosis Read More »

Did you know that CRF is the largest fund provider of cystinosis research in the world?  We have funded over 206 multi-year grants in 12 countries. When we started in 2003 there were a handful of researchers studying cystinosis but because of your support, we now have hundreds of researchers working every day to find …

CRF Research Insights – Your Donations at Work! Read More »

Did you know that your donations are at work all year long?  CRF awards new grants twice a year to ensure that your donations are always at work funding important research studies that will lead to a cure for cystinosis. Once a grant is awarded, grant payments are made upon receipt of a progress report …

CRF Research Insights – Your Donations Are Working for a Cure Read More »

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 …

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

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 …

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