Clinical Trial Update: An Open-label Safety, Pharmacokinetic, and Efficacy Study of Miglustat for the Treatment of Subjects With Batten Ceroid Lipofuscinosis, Neuronal 3 (CLN3) Disease [Phase 1, Phase 2]

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Summary

Open-label Phase 1/2 study evaluating the safety, pharmacokinetics, and efficacy of miglustat in Batten disease due to CLN3; the trial has completed with result…

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Diseases / indications

• Batten Disease
• Cln3 Disease

Drugs mentioned

• Miglustat (Zavesca) (Miglustat)

Snippet

Sponsor: Beyond Batten Disease Foundation | Conditions: Batten Disease | Phase 1, Phase 2 | Status: COMPLETED | Results posted: 2025-09-09