Treatment of the neuronal ceroid lipofuscinoses, also known as Batten disease, is at the start
of a new era because of diagnostic and therapeutic advances relevant to this group of …

Batten disease (also known as neuronal ceroid lipofuscinoses) constitutes a family of
devastating lysosomal storage disorders that collectively represent the most common …

The main aim of this review is to summarize the current state-of-art in the field of childhood
Neuronal Ceroid Lipofuscinosis (NCL), a group of rare neurodegenerative disorders. These …

The neuronal ceroid lipofuscinoses (NCLs) are a group of inherited neurodegenerative
lysosomal storage disorders (LSDs), traditionally grouped together based on shared clinical …

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

Neuronal ceroid lipofuscinoses (NCLs) are a group of rare, inherited, neurodegenerative
lysosomal storage disorders that affect children and adults. They are traditionally grouped …

Neuronal ceroid lipofuscinoses (Batten disease) are a group of inherited lysosomal storage
disorders characterized by progressive neurodegeneration leading to motor and cognitive …

Background Cerliponase alfa is a recombinant human tripeptidyl peptidase 1 (TPP1)
enzyme replacement therapy for the treatment of neuronal ceroid lipofuscinosis type 2 …

Late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease (Batten disease) is a
rare pediatric disease, with symptom development leading to clinical diagnosis. Early …

Introduction Neuronal ceroid lipofuscinosis type 2 (CLN2 disease) is a rare pediatric
neurodegenerative condition, which is usually fatal by mid-adolescence. Seizures are one of …