Neuronal Ceroid Lipofuscinosis Type
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CLINICAL_TRIALS trial_result · 2025-12-18Clinical Trial Update: A First-in-Human, Open-Label, Dose-Escalation Study to Evaluate the Safety and Tolerability of Gene Therapy With TTX-381 for the Ocular Manifestations Associated With Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) Disease [Phase 1, Phase 2]
First-in-human, open-label, dose-escalation trial assessing the safety and tolerability of the gene therapy TTX-381 for ocular manifestation…