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. website.

Nagamani SCS, Campeau PM, Shchelochkov OA, et al. Nitric-Oxide Supplementation for Treatment of Long-Term Complications in Argininosuccinic Aciduria. Am J Hum Genet. 2012;90(5):836-846.

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