Neuronal Ceroid Lipofuscinosis Type

Top drugs studied for this disease

All Approvals Safety alerts Label changes Trial results Research Guidance Withdrawals

CLINICAL_TRIALS research · 2025-12-31
Clinical Trial Update: Study for the Treatment for CLN7 Disease [Phase 1]
Phase 1 clinical trial for neuronal ceroid lipofuscinosis type 7 (CLN7) is listed as active but not recruiting. The sponsor is Benjamin Gree…
CLINICAL_TRIALS trial_result · 2025-12-18
Clinical Trial Update: A First-in-Human, Open-Label, Dose-Escalation Study to Evaluate the Safety and Tolerability of Gene Therapy With TTX-381 for the Ocular Manifestations Associated With Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) Disease [Phase 1, Phase 2]
First-in-human, open-label, dose-escalation trial assessing the safety and tolerability of the gene therapy TTX-381 for ocular manifestation…
CLINICAL_TRIALS research · 2025-06-11
Clinical Trial Update: Intravitreal ERT to Prevent Retinal Disease Progression in Children With CLN2 [Phase 1, Phase 2]
A Phase 1/2 clinical trial evaluating intravitreal enzyme replacement therapy to prevent retinal disease progression in children with Neuron…
CLINICAL_TRIALS research · 2024-11-15
Clinical Trial Update: A Multicenter Selective Screening Study to Investigate the Frequency of Neuronal Ceroid Lipofuxinosis Type 2 (CLN2)
A multicenter screening study is planned to determine the frequency of neuronal ceroid lipofuscinosis type 2 (CLN2). The study is currently …
FDA approval · 2024-07-10
FDA Approved: BRINEURA — Supplemental Application (BioMarin Pharmaceutical Inc.)
FDA approved BioMarin's supplemental application for BRINEURA (cerliponase alfa), updating the approved labeling for the product. This refle…
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FDA approval · 2018-12-11
FDA Approved: BRINEURA — Supplemental Application (BioMarin Pharmaceutical Inc.)
The FDA approved a supplemental application for BRINEURA (cerliponase alfa) from BioMarin Pharmaceutical Inc., updating the drug’s approved …
FDA approval · 2017-04-27
FDA Approved: BRINEURA — Original Application (BioMarin Pharmaceutical Inc.)
FDA approves Brineura (cerliponase alfa) through the original Biologics License Application by BioMarin for the treatment of neuronal ceroid…