Ornithine Transcarbamylase (OTC) Deficiency Clinical Trial Opportunities

Patient with Ornithine Transcarbamylase (OTC) Deficiency Patient with Ornithine Transcarbamylase (OTC) Deficiency

Learn how you or a loved one might participate in research studies

About Ornithine Transcarbamylase (OTC) Deficiency

OTC deficiency is the most common urea cycle disorder and leads to increased levels of ammonia. Patients with OTC deficiency suffer from acute hyperammonemic episodes that can lead to hospitalization, adverse cognitive and neurological effects, and potentially death.

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Who is Ultragenyx?

At Ultragenyx, our goal is to bring novel therapies to patients with serious rare and ultra-rare genetic diseases. Since our founding in 2010, our team has had a proven track record of identifying, developing, and launching medicines for rare diseases. During this time, we’ve launched 3 FDA-approved treatments in 4 indications and developed a diverse portfolio of investigational therapies. We have deep expertise in multiple therapeutic areas, such as endocrine/bone diseases, metabolic diseases, and central nervous system (CNS)/muscle diseases. Most importantly, we put patients and their caregivers at the center of our work. All our efforts are rooted in our commitment to provide patients and their families with hope today.

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Why are clinical trials important?

Clinical trials help provide invaluable information to empower healthcare professionals to treat, diagnose, or discover new breakthroughs in treating disease. By choosing to participate in a clinical trial, people may help advance treatments, provide crucial insights, and ultimately improve the quality of life for others. A person might also benefit in learning more about their condition, gain access to specific care, or achieve faster access to treatment.

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