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‹ Tue · 7 Jul 2026
Promising but preliminary

A Phase 1 Study of Convection-Enhanced Delivery of Intraputaminal AAV2-GDNF in Advanced Parkinson's Disease.

BACKGROUND: Parkinson's disease (PD) is a progressive neurodegenerative disorder. RESULTS: Across follow-up, 562 adverse events were recorded; 45 were considered possibly or probably related to the study drug.

BACKGROUND: Parkinson's disease (PD) is a progressive neurodegenerative disorder. RESULTS: Across follow-up, 562 adverse events were recorded; 45 were considered possibly or probably related to the study drug.

What the study was

Study design
Phase 1 clinical trial
Category
Drug Development
Maturity
Validated
Journal
Movement disorders : official journal of the Movement Disorder Society

Why it surfaced

Relevant Rare diseases with high unmet need study (promising preliminary signal) meets standard-priority threshold.

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