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R

RARE

Ultragenyx Pharmaceutical
Rare DiseaseScore: 79/100📋 Full Profile
B
79
Analyst Summary
Verified 2026-04-11

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].

Sources

  1. ClinicalTrials.gov (2026-04-11)
  2. ClinicalTrials.gov (2026-04-11)
  3. ClinicalTrials.gov (2026-04-11)
  4. ClinicalTrials.gov (2026-04-11)
  5. ClinicalTrials.gov · NCT05768854 (2026-02-25)
  6. ClinicalTrials.gov · NCT05933200 (2026-04-07)
  7. ClinicalTrials.gov · NCT05345171 (2026-02-17)
  8. SEC EDGAR · 0001515673 (2026-04-11)
Not financial advice. BiotechSigns scores are for informational purposes only and do not constitute investment recommendations. Always do your own due diligence before making investment decisions.
No active signals for RARE
Signal Summary
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Data Sources
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SEC EDGAR● LIVE
ClinicalTrials● LIVE
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