Trial Profile

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: 22 Oct 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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