Rebisufligene Etisparvovec

2 trials 2 recruiting Orphan designation

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2 Registered trials

2 Recruiting

0 Completed trials

1 Sponsors

1 Countries

Phase III

Phase I (FiH)

Phase III: 1 Phase I (FiH): 1

Indications tested

Mucopolysaccharidosis III 2

Sponsors using this IMP

Ultragenyx Pharmaceutical Inc. 2

All trials

2 total

A Long-term Follow-up Study of Patients with MPS IIIA from Gene Therapy Clinical Trials Involving the Administration of ABO-102 (scAAV9.U1a.… Recruiting

2023-510392-66-00 Phase III 1 country Ultragenyx Pharmaceutical Inc.
Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH for Mucopolysaccharidosis (MPS) IIIA Recruiting

2023-510032-37-00 Phase I (FiH) 1 country Ultragenyx Pharmaceutical Inc.

Showing 1–2 of 2 trials

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