Wilson disease


Is there a cure for Wilson disease?

A cure for Wilson disease would be a one-time treatment to fix the underlying cause of disease and prevent illness from occurring. Currently, we have life-long treatments but do not have a cure for Wilson disease.

There is current research being done to help develop a cure. One potential drug, called Curcumin, may help to partially turn back on the nonworking ATP7B genes in patients with Wilson disease. Turning the genes back on would allow patients to produce the missing protein that helps the body process copper and fix the underlying cause.

To find additional clinical research or current trials for Wilson disease, search clinicaltrials.gov. For private clinical trials, please visit www.centerwatch.com.

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What surveillance and monitoring is required for individuals with Wilson disease?

Is there a treatment for Wilson disease?

What surveillance and monitoring is required for individuals with Wilson disease?

Patients receiving copper chelation therapy should receive physical exams and routine tests to check liver health, blood counts and copper levels. It is important to follow these patients closely to ensure that the therapy type and dosage is effective, to monitor for adverse events, and to make sure that patients are complying with the doctors' orders and taking medications the right way. It's also important to make sure that copper levels do not drop too low. Copper deficiency can cause anemia, problems with the immune system, and neurological or brain-related symptoms. Excessive or long-term treatment can sometimes cause copper deficiency.

Other routine monitoring for Wilson disease patients includes biannual blood work to test liver health, biannual blood work to check blood counts, and biannual physical exam. Patients will also have a yearly 24-hour urine test to check copper levels in the urine.

Is there a treatment for Wilson disease?

Treatment for Wilson disease is a life-long process and involves reducing excess copper levels as well as preventing copper levels from building back up. It is often started as soon as possible in patients with Wilson disease who already have symptoms. Treatment helps to prevent serious damage to the liver and nervous system, reverse symptoms that have already started and improve emotional health.

The main form of treatment is called copper chelation therapy. Chelators are drugs that bind to excess copper in the body and help the body excrete copper through the urine. Common chelation drugs used to treat Wilson disease are d-Penicillamine and Trientine. Another treatment is taking zinc in high doses. Some zinc blocks the absorption of copper from the food we eat by interfering blocking absorption in the gastrointestinal tract. Zinc helps to reduce levels of copper and prevent copper from building back up. Zinc is most effective after the initial treatment with chelation therapy. Vitamin E is sometimes used with chelation or zinc therapy to protect tissue damage in the liver. In some patients, liver transplants may be necessary.

Doctors recommend that people with Wilson's disease limit copper in their diet by avoiding liver, shellfish, mushrooms, nuts and chocolate. Wilson disease patients should also not drink water from copper pipes and not take multivitamins or nutritional drinks, such as Ensure, containing copper.


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