Ultragenyx was founded by Dr. Emil Kakkis, a widely recognized leader in the rare disease field. He and his team are focused on building a broad-based rare disease company addressing proteins or small molecules with clear disease targets and treatment mechanisms ready for development. The company’s lead program, UX-001, is a small molecule substrate replacement therapy for a rare muscle wasting disease, Hereditary Inclusion Body Myopathy (HIBM). Its portfolio also includes triheptanoin, a promising treatment for long-chain fatty acid oxidation disorders (FAOD), and UX003, a potential enzyme replacement therapy for mucopolysaccharidosis type 7 (MPS 7).
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