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  • News bot March 12, 2026, 12:46 p.m.

    📋 ULTRAGENYX PHARMACEUTICAL INC. (RARE) - Clinical Trial Update

    Filing Date: 2026-03-12

    Accepted: 2026-03-12 08:45:25

    Event Type: Clinical Trial Update

    Event Details:

    ULTRAGENYX PHARMACEUTICAL INC. (RARE) Announces Clinical Trial Update ULTRAGENYX PHARMACEUTICAL INC. (RARE) provided an update on its clinical development programs. Clinical Development Highlights:
    • Collaboration: Leveroni Court
      • expected in the first half of 2027

    🔬 Clinical Development Pipeline (ULTRAGENYX PHARMACEUTICAL INC.):

    Product Type Development Stage Therapeutic Area Source
    No Investigational Product OTHER Phase PHASE3 Mucopolysaccharidosis IIIA ClinicalTrials.gov
    Adjuvant Immunomodulatory (IM) Therapy DRUG Phase PHASE3 Mucopolysaccharidosis IIIA ClinicalTrials.gov
    DTX301 GENETIC Phase PHASE3 OTC Deficiency ClinicalTrials.gov
    Placebo for oral prednisolone DRUG Phase PHASE3 Glycogen Storage Disease Type IA ClinicalTrials.gov
    Oral prednisolone DRUG Phase PHASE3 Glycogen Storage Disease Type IA ClinicalTrials.gov
    Placebo OTHER Phase PHASE3 Glycogen Storage Disease Type IA ClinicalTrials.gov
    DTX401 GENETIC Phase PHASE3 Glycogen Storage Disease Type IA ClinicalTrials.gov
    No Intervention OTHER Preclinical Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) ClinicalTrials.gov
    Triheptanoin DRUG Phase PHASE2 Medium-chain Acyl-CoA Dehydrogenase Deficiency ClinicalTrials.gov
    GTX-102 DRUG Phase PHASE2 Angelman Syndrome ClinicalTrials.gov
    BAY2599023 (DTX201) DRUG Phase PHASE1 Hemophilia A ClinicalTrials.gov
    Placebo (for Pitavastatin) DRUG Phase PHASE4 Hypercholesterolemia ClinicalTrials.gov
    Placebo (for Pitavastatin/Ezetimibe) DRUG Phase PHASE4 Hypercholesterolemia ClinicalTrials.gov
    Ezetimibe 10 mg DRUG Phase PHASE4 Hypercholesterolemia ClinicalTrials.gov
    Pitavastatin 4mg DRUG Phase PHASE4 Hypercholesterolemia ClinicalTrials.gov
    Empagliflozin/Linagliptin DRUG Phase PHASE4 Type 2 Diabetes Mellitus (T2DM) ClinicalTrials.gov
    Metformin DRUG Phase PHASE4 Type 2 Diabetes Mellitus (T2DM) ClinicalTrials.gov
    Anagliptin DRUG Phase PHASE4 Type 2 Diabetes Mellitus (T2DM) ClinicalTrials.gov
    Empagliflozin/Metformin DRUG Phase PHASE4 Type 2 Diabetes Mellitus (T2DM) ClinicalTrials.gov
    Setrusumab BIOLOGICAL Phase PHASE2 Osteogenesis Imperfecta ClinicalTrials.gov
    Bisphosphonate DRUG Phase PHASE3 Osteogenesis Imperfecta ClinicalTrials.gov
    Sodium Acetate DRUG Phase PHASE3 OTC Deficiency ClinicalTrials.gov
    Placebo for oral corticosteroids DRUG Phase PHASE3 OTC Deficiency ClinicalTrials.gov
    Oral Corticosteroids DRUG Phase PHASE3 OTC Deficiency ClinicalTrials.gov
    Sham-LP PROCEDURE Phase PHASE3 Angelman Syndrome ClinicalTrials.gov
    Mepsevii DRUG Preclinical MPS VII ClinicalTrials.gov
    Venipuncture PROCEDURE Preclinical Mucopolysaccharidoses ClinicalTrials.gov
    CWP232291 DRUG Phase PHASE1 Acute Myeloid Leukemia ClinicalTrials.gov
    Adjuvant IM Therapy DRUG Phase PHASE2 MPS IIIA ClinicalTrials.gov
    Optimized Prophylactic IM Therapy DRUG Phase PHASE2 MPS IIIA ClinicalTrials.gov
    Prophylactic Immunomodulatory (IM) Therapy DRUG Phase PHASE2 MPS IIIA ClinicalTrials.gov
    UX111 BIOLOGICAL Phase PHASE2 MPS IIIA ClinicalTrials.gov
    Standard of Care (SOC) DRUG Phase PHASE1 Wilson Disease ClinicalTrials.gov
    UX701 GENETIC Phase PHASE1 Wilson Disease ClinicalTrials.gov
    GLUT1 DS DRUG Phase PHASE2 Glut1 Deficiency Syndrome ClinicalTrials.gov

    💼 Business Developments:

    • Partnership: Not available
    • Acquisition: Not available
    • Licensing: Not available
    • Regulatory Approval
    • Executive Changes: Not available

    Structured Data:

    • Company Name: ULTRAGENYX PHARMACEUTICAL INC.
    • Ticker Symbol: RARE

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