Breakthrough Cystinosis Cure Is On the Way!

On a press release reported from BusinessWire, the Cystinosis Research Foundation, CRF, awarded unparalleled grants of $1,022,879 to research institutions making it possible to further investigate innovative treatments for cystinosis. With a cystinosis cure being the end goal, scientists are carefully studying this rare metabolic and genetic disease.

Cystinosis affects just about 500 young adults and children in the United States and approximately 2,000 people globally.

Sadly, as cystinosis progresses, it destroys every organ in the body. The disease ravages the liver, kidneys, muscles, eyes, the thyroid, and the brain. Currently, there is one treatment, cysteamine, which is proven to help extend the lives of patients, but the cystinosis cure remains a top priority for scientists who work tirelessly to make this dream a reality. Now more than ever, this is why the funding for additional research is so crucial.

The press release details that several proposals that received a CRF semi-annual grant were for new areas of research in clinical trials for the development of new therapies. This has the cystinosis community excited believing they are on the brink of breakthrough treatments, which could potentially, be made affordable with financial support services from pharmaceutical companies.

Cystinosis magazine
Although the Cystinosis Research Foundation is well-known for funding breakthroughs toward a cystinosis cure, they also share personal stories, cystinosis news, and a host of information in the Cystinosis Magazine. Check out the latest issue.

Under the leadership of the CRF’s Scientific Review Board, renowned cystinosis researchers and scientists evaluate findings from clinical trials and leading research. With this in mind, every dollar raised by the CRF is committed to medical research for finding a cystinosis cure. Administrative costs are privately underwritten.

Below is a list of recipients awarded grants in 2015 by the Cystinosis Research Foundation Totaling: $1,022,879

Ghanashyam Acharya, PhD, Principal Investigator Baylor College of Medicine, Houston, Texas, “Development of Transdermal Cysteamine Delivery System” Award – $112,500

Stéphanie Cherqui, PhD, Principal Investigator University of California, San Diego, “Mechanism of Bone Marrow Stem Cell-Mediated Therapy in the Mouse Model of Cystinosis” Award – $364,800

Liang Feng, PhD, Mentor, Xue Guo, PhD, Fellow, Stanford University, Palo Alto, California, “Molecular Mechanism of Cystinosis” Award – $225,000

Sihoun Hahn, MD, PhD, Principal Investigator, Seattle Children’s Hospital, Seattle, Washington, “Newborn Screening for Cystinosis” Award – $153,929

Anna Taranta, PhD, Principal Investigator, Andrea Del Fattore, PhD, Co-Principal Investigator, Bambino Gesù Children’s Hospital, Rome, Italy, “A Study on Bone Remodeling Defects in Nephropathic Cystinosis” Award – $166,650

To learn more about the organization’s next steps, check out the foundation’s homepage.


Do you think a million bucks is enough or do you want to see more done for those living with cystinosis? Be apart of the solution by sharing this article and pushing for more funding for a cystinosis cure!

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