Late-infantile Neuronal Ceroid Lipofuscinosis
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FDA approval · 2024-07-24FDA Approved: BRINEURA — Supplemental Application (BioMarin Pharmaceutical Inc.)
The FDA approved a supplemental application for BRINEURA (cerliponase alfa) submitted by BioMarin Pharmaceutical Inc.
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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 · 2015-01-15Clinical Trial Update: Study of HuCNS-SC Cells in Patients With Infantile or Late Infantile Neuronal Ceroid Lipofuscinosis (NCL) [Phase 1]
A Phase 1 study evaluating HuCNS-SC neural stem cells in infants with infantile or late-infantile neuronal ceroid lipofuscinosis has complet…
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