GBA1-related Parkinson disease, susceptibility
MONDO:1040030A susceptibility or predisposition to Parkinsons disease in which the cause of the disease is the presence of a monoallelic pathogenic variant or risk allele in the GBA1 gene.
6 clinical trials for this condition and its sub-types, 6 tagged with GBA1-related Parkinson disease, susceptibility itself.
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Free drug access for Parkinson's patients with specific gene mutation
Disease control Expanded access (ended)This program offers continued free access to the experimental drug BIA 28-6156 for people with Parkinson's disease who have a specific change in the GBA1 gene and have already completed the ACTIVATE study. Participants take one 60mg pill daily. The goal is to provide ongoing trea…
Sponsor: Bial R&D Investments, S.A. • Aim: Disease control
Last updated Aug 12, 2026 00:00 UTC
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Gene therapy trial targets Parkinson's at its genetic root
Disease control CancelledThis study tests a one-time gene therapy called CAP-003 in adults with Parkinson's disease caused by a GBA1 gene mutation. The goal is to see if it is safe and if it can help control the disease. Participants receive a single IV infusion and are monitored for two years.
Phase 1/2 • Sponsor: Capsida Biotherapeutics, Inc. • Aim: Disease control
Last updated Jun 27, 2026 14:01 UTC
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New pill aims to slow Parkinson's in patients with genetic mutation
Disease control OngoingThis study tests an experimental drug called BIA 28-6156 in people with Parkinson's disease who have a specific change in the GBA1 gene. The goal is to see if the drug can delay the worsening of movement-related daily living skills over 78 weeks. About 237 participants will recei…
Phase 2 • Sponsor: Bial R&D Investments, S.A. • Aim: Disease control
Last updated Jun 27, 2026 12:34 UTC