Ultragenyx: Clinical Trial for OTC

Who are Ultragenyx?

Ultragenyx is an American biopharmaceutical company involved in the research and development of new products for treatment of rare and ultra-rare genetic diseases for which there are typically no approved treatments and high unmet medical need.

What work is Ultragenyx doing?

DTX301 is an investigational gene therapy that is being developed for the treatment of ornithine transcarbamylase (OTC) deficiency. The purpose of this study is to determine the efficacy and confirm the safety of DTX301 in patients 12 years of age and older with late-onset OTC deficiency.

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