Uniqure Biopharma B.v.
Clinical trials sponsored by Uniqure Biopharma B.v., explained in plain language.
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Gene therapy injection aims to quiet seizures in Drug-Resistant epilepsy
Disease control Recruiting nowThis early-stage study tests a gene therapy called AMT-260 in 12 adults with a specific type of epilepsy (mesial temporal lobe epilepsy) that does not respond to standard medications. The treatment is delivered directly into the brain using MRI guidance. The main goals are to che…
Phase: PHASE1, PHASE2 • Sponsor: UniQure Biopharma B.V. • Aim: Disease control
Last updated May 26, 2026 08:54 UTC
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Gene therapy trial hopes to tame rare enzyme disorder
Disease control Recruiting nowThis study tests a single-dose gene therapy called AMT-191 in 12 adult men with classic Fabry disease. The goal is to see if the treatment is safe and how it works in the body. Fabry disease is a rare genetic condition that can cause pain, organ damage, and other serious problems…
Phase: PHASE1, PHASE2 • Sponsor: UniQure Biopharma B.V. • Aim: Disease control
Last updated May 26, 2026 08:41 UTC