Ultragenyx Pharmaceutical

Ultragenyx Pharmaceutical (RARE) is the lead sponsor of 17 active clinical trials listed on ClinicalTrials.gov^[4], including 9 Phase 3^[1], 4 Phase 2^[2], 1 Phase 1^[3].

Trial NCT05768854^[5] evaluates Bisphosphonate in Osteogenesis Imperfecta with a target enrollment of 69 participants. Trial NCT05933200^[6] evaluates Triheptanoin in Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) with a target enrollment of 69 participants. Trial NCT05345171^[7] evaluates DTX301 in OTC Deficiency with a target enrollment of 32 participants.

RARE has 1 Form 4 insider filing recorded at the SEC in the past 30 days^[8].