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A Long-term Follow-up Study of Patients With MPS IIIA From Gene Therapy Clinical Trials Involving the Administration of ABO-102 (scAAV9.U1a.hSGSH)

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A Long-term Follow-up Study of Patients With MPS IIIA From Gene Therapy Clinical Trials Involving the Administration of ABO-102 (scAAV9.U1a.hSGSH)

Status: Recruiting
Phase of Trial: Phase III

Latest Information Update: 07 Feb 2025

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At a glance

  • Drugs Rebisufligene etisparvovec (Primary)
  • Indications Mucopolysaccharidosis III
  • Focus Adverse reactions; Registrational; Therapeutic Use
  • Sponsors Abeona therapeutics; Ultragenyx Pharmaceutical

    • Most Recent Events

    • 05 Feb 2025 According to an Ultragenyx Pharmaceutical media release, data from this study will be presented at the WORLDSymposium 2025 21st Annual Research Meeting, taking place February 3-7 in San Diego.
    • 15 Jan 2025 Planned number of patients changed from 34 to 41.
    • 15 Jan 2025 Planned End Date changed from 1 Jun 2027 to 1 Aug 2027.

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