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Clinical Trials for Neuronal Ceroid-Lipofuscinoses

• An Open-label Safety, Pharmacokinetic, and Efficacy Study of the Combination of Trehalose and Miglustat for the Treatment of CLN3 Disease
• Intravitreal ERT to Prevent Retinal Disease Progression in Children With CLN2
• Safety, Tolerability, and Efficacy of PLX-200 in Patients With CLN3
• Batten CLN6 Gene Therapy
• A Safety, Tolerability, and Efficacy Study of Intracerebroventricular BMN 190 in Patients With CLN2 Disease
• Human Placental-Derived Stem Cell Transplantation
• Cellcept for Treatment of Juvenile Neuronal Ceroid Lipofuscinosis
• Study of HuCNS-SC Cells in Patients With Infantile or Late Infantile Neuronal Ceroid Lipofuscinosis (NCL)
• Stem Cell Transplant for Inborn Errors of Metabolism
• Cystagon to Treat Infantile Neuronal Ceroid Lipofuscinosis