Uniqure Biopharma B.v.
Clinical trials sponsored by Uniqure Biopharma B.v., explained in plain language.
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One-Time gene therapy aims to tame rare enzyme disorder
Disease control Recruiting nowThis early-stage study tests a single dose of a gene therapy called AMT-191 in 12 adult men with classic Fabry disease. The treatment uses a harmless virus to deliver a working copy of the GLA gene, which is faulty in Fabry disease. The main goals are to check safety and how the …
Phase: PHASE1, PHASE2 • Sponsor: UniQure Biopharma B.V. • Aim: Disease control
Last updated May 11, 2026 20:49 UTC
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Gene therapy injection aims to quiet seizures in Drug-Resistant epilepsy
Disease control Recruiting nowThis early-stage study tests a new 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…
Phase: PHASE1, PHASE2 • Sponsor: UniQure Biopharma B.V. • Aim: Disease control
Last updated May 11, 2026 20:37 UTC