Donate 5th Annual Dublin Cystinosis Workshop 2019 - 26 & 27 April 2019

Treatment of Cystinosis

Although there is no cure for cystinosis yet, there is a specific treatment available for the treatment of this disease.  The drug cysteamine bitartrate, known as Cystagon™, helps reduce cystine accumulation within the cells and has proven effective in delaying renal failure and improving growth.  It is not yet known whether this benefits all organs of the body as some complications of the disease appear much later than others.

Cysteamine treatment is only effective if taken every six hours, day and night for life. It does however have some potentially unpleasant side effects including nausea, vomiting, abdominal pain, body and breath odour. There are also some very rare and extremely serious side effects which can cause changes in bones and skin tissue. Any unusual clinical occurrences must be discussed with the patient’s physician.

There is also a slow release formulation of this drug, known as Procysbi™ which is only taken twice a day, every twelve hours. This is not yet available in Ireland, but we are working with pharma to help the drug application process to be submitted.

Eyedrops to remove corneal crystals, Cystadrops™, are given four times a day, every day.

Due to impaired kidney function children and adolescents need to take mineral supplements such as calcium, potassium, vitamin D, phosphate and sodium bicarbonate to replace those lost.

As the kidneys are the first organ to be severely affected, kidney function slowly diminishes and a transplant may be needed.  The transplanted kidney is not affected by Cystinosis.

Some children with poor growth may receive growth hormone treatments.

Some children may have difficulty swallowing, or experience abdominal pain.  They will be evaluated by a gastroenterologist and may require additional treatments or medications to help alleviate their symptoms.

Hope will find a cure

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