Details about studies in France and the Netherlands...

This study is currently recruiting participants.

This study will continue to evaluate the long term safety and effectiveness of cysteamine eye drops for treating cystine crystals in the corneas of patients with cystinosis. These drops are not sold commercially and are available only through this study. New patients may enroll in the study to obtain them....

This study is currently recruiting participants.

Cystinosis is an inherited disease resulting in poor growth and kidney failure. There is no known cure for cystinosis, although kidney transplantation may help the renal failure and prolong survival. Both the kidney damage and growth failure are thought to be due to the accumulation of the amino acid cystine within the cells of the body. The cystine storage later damages other organs besides the kidneys, including the thyroid gland, pancreas, eyes, and muscle.

This study is currently recruiting participants.

This is a long-term, open-label, study to determine the safety and tolerability of twice a day treatment with Cysteamine Bitartrate Delayed-release Capsules(RP103).

Desperate for answers, she turned to experts. After two months, doctors diagnosed Allison with cystinosis, a rare genetic disease that causes kidney failure, usually by age 9.

On a global level, the efforts of local individuals working to help Hank already have gained notice from the Cystinosis Research Foundation — an organization that is pressing for a cure to the disease.

The success of this year's local Real Men Can Cook event has enabled the organizers of the fundraiser to present a cheque to the Cystinosis Research Foundation for $30,334.

Morven, daughter of Alex and Neil Hutchison, was diagnosed with Cystinosis at 9 months old. Since the diagnosis the family, who live in West Linton, south of Edinburgh, have been very keen to support the Cystinosis Foundation UK.

Dan, aided by a new kidney transplanted in January to replace the ones wracked by cystinosis, walked with 620 other seniors to receive his Liberty High School diploma in a ceremony neither he nor his parents or teachers thought he'd be able to attend.

Now, the 20-year-old, of Moorside, Sunderland, is praying for a miracle as she waits for a suitable donor. Sarah has to endure three hours of dialysis four times a week at Newcastle’s Royal Victoria Infirmary.

At just 18 months old, the youngster was diagnosed with cystinosis, a rare genetic disease that affects just one in 200,000 people.

From Wall St. Journal

This study is currently recruiting participants.

Cure Cystinosis International Registry (CCIR) is an online, patient self-identifying registry developed by medical and scientific experts specifically for the cystinosis community.

CCIR's sole purpose is to identify people with cystinosis worldwide in an effort to accelerate novel treatments and a cure for cystinosis.

CCIR provides a safe and secure platform for: sharing anonymous medical information about cystinosis with researchers, clinicians and patients, disseminating information about research opportunities, and connecting researchers/investigators and prospective participants.

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As part of our continuing work to obtain new pro-drugs for the treatment of nephropathic cystinosis, a number of glutaric and succinic acid derivatives of cystamine have been designed, synthesised and biologically evaluated in vitro. These compounds have been designed as odourless and tasteless...

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