Clinical Trial Update: A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 190 in Patients With Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN2) Disease [Phase 1, Phase 2]

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Summary

Phase 1/2 open-label dose-escalation trial evaluating intracerebroventricular BMN 190 for safety, tolerability, PK, and efficacy in CLN2 disease; study complete…

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Diseases / indications

• Batten Disease
• Cln2 Disease
• Jansky-bielschowsky Disease
• Late-infantile Neuronal Ceroid Lipofuscinosis

Drugs mentioned

• BMN 190

Snippet

Sponsor: BioMarin Pharmaceutical | Conditions: Jansky-Bielschowsky Disease, Batten Disease | Phase 1, Phase 2 | Status: COMPLETED | Results posted: 2018-06-11