Late-infantile Neuronal Ceroid Lipofuscinosis Type
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CLINICAL_TRIALS research · 2026-01-14Clinical Trial Update: Cerliponase Alfa Observational Study in the US
An update on an observational US study of cerliponase alfa for late-infantile neuronal ceroid lipofuscinosis type 2, noting the study is act…
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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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FDA approval · 2020-03-27FDA Approved: BRINEURA — Supplemental Application (BioMarin Pharmaceutical Inc.)
FDA approves a supplemental application for Brineura (cerliponase alfa) from BioMarin Pharmaceutical Inc.