Batten Disease
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CLINICAL_TRIALS trial_result · 2025-09-09Clinical 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]
Open-label Phase 1/2 study evaluating the safety, pharmacokinetics, and efficacy of miglustat in Batten disease due to CLN3; the trial has c…
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CLINICAL_TRIALS trial_result · 2025-07-30Clinical Trial Update: Natural History Study of Batten Disease
The natural history study of Batten disease (neuronal ceroid lipofuscinosis CLN6/CLN3) has been terminated. No further results are expected …
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CLINICAL_TRIALS trial_result · 2025-02-17Clinical Trial Update: A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease [Phase 2]
Phase 2 safety, tolerability, and efficacy study of BMN 190 (cerliponase alfa) in pediatric patients under 18 with CLN2 disease has complete…
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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 · 2020-08-31Clinical Trial Update: AAVRh.10 Administered to Children With Late Infantile Neuronal Ceroid Lipofuscinosis [Phase 1, Phase 2]
Clinical trial update on AAVRh.10 administration in children with Late Infantile Neuronal Ceroid Lipofuscinosis (Batten disease) spanning Ph…
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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…
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CLINICAL_TRIALS trial_result · 2016-07-29Clinical Trial Update: Genotype - Phenotype Correlations of LINCL
This clinical trial update reports on genotype-phenotype correlations in LINCL, a form of Batten disease, sponsored by Weill Medical College…
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CLINICAL_TRIALS trial_result · 2016-07-26Clinical Trial Update: Neuronal Ceroid Lipofuscinosis and Associated Sleep Abnormalities
A completed clinical trial sponsored by Nationwide Children's Hospital studied sleep abnormalities in people with Neuronal Ceroid Lipofuscin…