Orphanet: Cystinosis European Clinical Trials
Google BlogSearch, 2011-04-21 00:00:00

Details about studies in France and the Netherlands...

Cysteamine Eye Drops to Treat Corneal Crystals in Cystinosis
Clinical Trials - Muckle Wells, 2011-03-29 00:00:00

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....

Use of Cysteamine in the Treatment of Cystinosis
Clinical Trials - Muckle Wells, 2011-03-01 00:00:00

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.

Long-Term Safety Follow-up Study of Cysteamine Bitartrate Delayed-release Capsules (RP103)
Clinical Trials - Muckle Wells, 2011-07-21 00:00:00

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).

Payin’ it forward: Racer goes online to battle Cystinosis
Google BlogSearch, 2011-07-18 00:00:00

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.
Google BlogSearch, 2011-07-18 00:00:00

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.

Mother Donates Kidney to Save Daughter's Life
Google BlogSearch, 2011-05-09 00:00:00

It took doctors a while to figure it out, but at age 4, she was diagnosed with Cystinosis - a genetic disorder that affects the kidneys. She would eventually need a kidney transplant. Doctors looked for a donor in her family....

Graduating with a diploma, new kidney
Google BlogSearch, 2011-06-21 00:00:00

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.

Sarah’s race for life as rejection leaves her waiting for second kidney transplant
Google BlogSearch, 2011-05-15 00:00:00

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.

Cure Cystinosis International Registry (CCIR)
Google BlogSearch, 2010-08-01 00:00:00

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.

Synthesis and in vitro evaluation of novel pro-drugs for the treatment of nephropathic cystinosis.
Recent scientific publications, 2011-05-10 00:00:00

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...