Article

Understanding the Patient Journey, Current Treatment Approaches and Emerging Therapeutic Opportunities in CLN2 Batten Disease

2026-06-02

Abstract excerpt

<h4>ABSTRACT</h4> <h4>Background</h4> CLN2 disease, Neuronal Ceroid Lipofuscinosis (NCL) type 2, is a rare, genetic neurodegenerative condition predominantly affecting children. CLN2 disease is characterized by seizures, language and motor decline, vision loss, and premature death. Currently, the only regulatory-approved therapy is the enzyme replacement therapy (ERT) Cerliponase alfa, administered fortnightly v...

Topics

Open a Topic to create a Post that cites this publication.

Identifiers and source

Literature Corpus work: 26f8947a-7487-5b91-8595-258c9067ea6b
DOI: 10.64898/2026.05.31.26354557

Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest

Understanding the Patient Journey, Current Treatment Approaches and Emerging Therapeutic Opportunities in CLN2 Batten DiseaseDOI 10.64898/2026.05.31.26354557Read

Guidelines on the Diagnosis, Clinical Assessments, Treatment and Management for CLN2 Disease PatientsSemantic proximity Read

Management of CLN1 Disease: International Clinical Consensus.Semantic proximity Read

Cerebrospinal fluid neurofilament light chain levels in CLN2 disease patients treated with enzyme replacement therapy normalise after two years on treatmentSemantic proximity Read

Cerliponase alfa in the treatment of patients with classic and atypical late infantile neuronal ceroid lipofuscinosis in Latin AmericaSemantic proximity Read

A needle in a haystack? The impact of a targeted epilepsy gene panel in the identification of a treatable but rapidly progressive metabolic epilepsy: CLN2 disease.Semantic proximity Read

Towards understanding the neuronal ceroid lipofuscinoses.Semantic proximity Read

Neuronal Ceroid Lipofuscinosis—Concepts, Classification, and Avenues for TherapySemantic proximity Read

Classification and natural history of the neuronal ceroid lipofuscinoses.Semantic proximity Read

Neuronal ceroid lipofuscinosis: impact of recent genetic advances and expansion of the clinicopathologic spectrum.Semantic proximity Read

Therapeutic approaches to the challenge of neuronal ceroid lipofuscinoses.Semantic proximity Read

Clinical Characteristics and Treatment Experience of Individuals with SCN8A Developmental and Epileptic Encephalopathy (SCN8A-DEE): Findings from an Online Caregiver SurveySemantic proximity Read

Characterisation of early changes in ovine CLN5 and CLN6 Batten disease neural cultures for the rapid screening of therapeutics.Semantic proximity Read