GTX-102

About Ultragenyx Pharmaceutical

Ultragenyx is a leader in the rare disease space, with a mission to bring transformative treatments to patients with severe, life-altering genetic conditions. The company has successfully commercialized products such as Crysvita® (burosumab) for XLH and Dojolvi® for long-chain fatty acid oxidation disorders (LC-FAOD), while advancing a deep pipeline of gene therapy, mRNA, and other biologic candidates. Its strategy focuses on leveraging multiple technology platforms to address high-unmet-need diseases, supported by strategic partnerships and a global commercial footprint.

Therapeutic Areas

Other Angelman Syndrome Drugs