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181 results for “CLN1”

Trials

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Natural History and Longitudinal Clinical Assessments in NCL / Batten Disease, the International DEM-CHILD Database

500 participants · First posted Nov 3, 2020

Clinical and Neuropsychological Investigations in Batten Disease

500 participants · First posted Jun 10, 2013

Not yet recruiting

Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of PLX-200 in Pediatric Patients (Master Protocol)

24 participants · First posted Jul 31, 2026

Drugs

View all 54 →

CLN1 disease(Spark)

CLN1 gene therapy (Amicus Therapeutics)

Investigational

ABO-202

News

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NCL Translational Research Conference Highlights Advances in Batten Disease Therapies

FDA Grants Fast Track Designation to Alentis Therapeutics' ALE.P02 for CLDN1+ Squamous Cancers

FDA Expands Brineura Approval to Include All Ages with CLN2 Disease

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