Argininosuccinic aciduria

Clinical Research/Studies

Is there clinical research happening on argininosuccinic aciduria?

As of July 2017, there were two clinical trials specific to argininosuccinic aciduria listed on, a government website that provides regularly updated information about federally and privately supported clinical trials. Both studies are looking into nitric oxide as a potential therapy. The enzyme argininosuccinate lyase is reduced in people with this disorder and this enzyme helps to create nitric oxide in the body. Nitric oxide is a compound that has many different functions in the body.

Although there were only 2 clinical trials, there were 12 trials investigating urea cycle disorders as a group. Argininosuccinic aciduria is classified as a urea cycle disorder. For the most up-to-date list, contact clinical and search for argininosuccinic aciduria or urea cycle disorders.

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How do I find clinical research on argininosuccinic aciduria?

How do I find clinical research on argininosuccinic aciduria?

To learn about ongoing clinical research into argininosuccinic aciduria ask a physician who specializes in the disorder. Genetic counselors and medical geneticists are also excellence sources of information about ongoing clinical trials and can offer advice about the pros and cons about participating in such a trial. A medical geneticist can be found by asking your doctor for a referral or looking on the American College of Medical Geneticists website. Genetic counselors in the United States can be found on the National Society of Genetic Counselors website. Genetic counselors in Canada can be found at the Canadian Association of Genetic Counselors website

Ongoing studies for argininosuccinic aciduria can also be found on, a service of the U.S. National Institutes of Health. In Europe, the EU Clinical Trial Register performs a similar service.

Support groups also have information about ongoing clinical trials as well as information and advice about participating in clinical trials. Support groups for argininosuccinic aciduria include the National Urea Cycle Disorders Foundation.

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