Research Progress Update by Morgan DiLeo, PhD, University of Pittsburgh
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 […]
The Third Patient Was Transplanted November 16, 2020!
From Rocky Seachord, Jacob’s Mom: “He is in very good spirits and I think it is a good omen that he is in the very same room that Jordan was in… The transplant, considered Day 0, is Monday the 16th. This will be his new birthday. I can’t even imagine the possibilities in his life […]
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 […]
Stem Cell And Gene Therapy Webinar Update Video Now Available!
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, […]
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 […]
CRF Research Insights – Your Donations at Work!
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 Are Working for a Cure
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 […]
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 […]
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 […]
JOIN OUR JOURNEY TO A CURE ON RARE DISEASE DAY!
There are approximately 7,000 rare diseases that affect 30 million people in the U.S. and 300 million people in the world, and only 5% have an FDA-approved treatment. Cystinosis affects approximately 2,000 people in the world, and we are fortunate to have a treatment. In fact, as a direct result of CRF’s funding and strategy […]