Jansky-bielschowsky Disease
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CLINICAL_TRIALS trial_result · 2022-08-24Clinical Trial Update: An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease [Phase 1, Phase 2]
An extension study assessed long-term efficacy and safety of BMN 190 in patients with CLN2 disease (a form of Batten disease), reporting res…
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CLINICAL_TRIALS trial_result · 2018-06-11Clinical 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]
Phase 1/2 open-label dose-escalation trial evaluating intracerebroventricular BMN 190 for safety, tolerability, PK, and efficacy in CLN2 dis…