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.
10 clinical trials for this condition and its sub-types.
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Gene therapy trial targets Parkinson's at its genetic root
Disease control TerminatedThis 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: PHASE1, PHASE2 • 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: PHASE2 • Sponsor: Bial R&D Investments, S.A. • Aim: Disease control
Last updated Jun 27, 2026 12:34 UTC
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Quick genetic test for unborn babies hits a snag: study withdrawn
Knowledge-focused TerminatedThis study aimed to see if a rapid whole genome sequencing test on amniotic fluid could give faster genetic diagnoses for fetuses with suspected genetic diseases. Researchers planned to compare results from amniotic fluid and blood samples from up to 90 families. However, the stu…
Sponsor: Mayo Clinic • Aim: Knowledge-focused
Last updated Jun 26, 2026 14:00 UTC