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What is the therapeutic window between GCS inhibition efficacy and systemic toxicity in GBA-PD patients?
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neurodegeneration
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The debate identified systemic toxicity as a major concern for glucosylceramide synthase inhibitors, but no dose-response data exists for PD patients. This critical safety gap must be resolved before clinical translation of substrate reduction therapy.
Source: Debate session sess_sda-2026-04-01-002 (Analysis: sda-2026-04-01-002)