Search Results - neuronal+ceroid-lipofuscinoses+(ncl)

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CNS Therapeutics That Target Neuronal Ceroid-Lipofuscinoses and Thioesterase Deficiency Disorders

Abstract: Clinically known as Neuronal Ceroid-Lipofuscinoses (NCL), Batten disease, is a rare neuron killing disease and one of the lysosomal storage disorders (LSDs). It is associated with a mutation or lack of palmitoyl-protein thioesterase-1 (PPT1) gene. It manifests very early in a child's life causing absence of brain activity as early as 4 years...

Published: 4/8/2024 | Inventor(s): Anil Mukherjee, Chinmoy Sarkar, Gary Zhongjian Zhang

Keywords(s): lysosomal storage disorders (LSD), lysosome, Neuronal Ceroid-Lipofuscinoses (NCL), Non-toxic derivative of hydroxylamine, Palmitoyl-protein thioesterase

Category(s): Application > Therapeutics, Collaboration Sought > Collaboration, Collaboration Sought > Licensing