CLN2 disease (neuronal ceroid lipofuscinosis type 2) is a rare, autosomal recessive,
pediatric-onset, rapidly progressive neurodegenerative lysosomal storage disorder caused …

Neuronal ceroid lipofuscinosis type 2 (CLN2 disease) is a progressive neurodegenerative
disease that results in early-onset, severe, progressive, neurological disabilities, leading to …

Late infantile Batten disease (CLN2 disease) is an autosomal recessive, neurodegenerative
lysosomal storage disease caused by mutations in the CLN2 gene encoding tripeptidyl …

Neuronal ceroid lipofuscinoses (NCLs) are a heterogeneous group of lysosomal storage
disorders. NCLs include the rare autosomal recessive neurodegenerative disorder neuronal …

Introduction The use of enzymes in various industries has been prevalent for centuries.
However, their potency as therapeutics remained latent until the late 1950 s, when scientists …

Abstract Background CLN2 disease (Neuronal Ceroid Lipofuscinosis Type 2) is an ultra-
rare, neurodegenerative lysosomal storage disease, caused by an enzyme deficiency of …

Aim Late infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease is a rare
neurodegenerative disorder presenting in children aged 2–4 years with seizures and loss of …

Abstract Background/Objectives CLN2 Batten Disease is a fatal neurodegenerative
condition of childhood associated with retinal dystrophy and blindness …

Conducting clinical trials in rare diseases is challenging. In trials that aim to use natural
history control cohorts for evaluation of efficacy, lack of data on natural history of disease …

Batten Disease (Juvenile Neuronal Ceroid Lipofuscinosis) - StatPearls - NCBI Bookshelf US
flag An official website of the United States government Here's how you know NIH NLM …