Sage Journals: Discover world-class research

Retricted access

Review article

First published 2026-1

CLN2 Disease: Current Understandings,Challenges,and Future Directions

Maria Shock, Elisa Nigro, Elizabeth J Donner [...]

Journal of Child Neurology

Open access

Research article

First published 2018

An Adapted Clinical Measurement Tool for the Key Symptoms of CLN2 Disease

Kathleen W. Wyrwich, Angela Schulz, Miriam Nickel [...]

Journal of Inborn Errors of Metabolism and Screening

Open access

Research article

First published 2021-5

Cerliponase Alfa for the Treatment of Atypical Phenotypes of CLN2 Disease: A Retrospective Case Series

Eva Wibbeler, Raymond Wang, Emily de los Reyes [...]

Journal of Child Neurology

Open access

Research article

First published 2021-7

Development of the “Hamburg Best Practice Guidelines for ICV−Enzyme Replacement therapy (ERT) in CLN2 Disease” Based on 6 Years Treatment Experience in 48 Patients

Christoph Schwering, Gertrud Kammler, Eva Wibbeler [...]

Journal of Child Neurology

Open access

Article commentary

First published 2023-12

Revoking the Seize Order: Preventing Spontaneous Seizures With AAV in a CLN2 Mouse Model

Gordon F. Buchanan

Epilepsy Currents

Retricted access

Other

First published 2007-5

Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN2/Jansky-Bielschowsky Type) in Oman

Roshan Koul, Amna Al-Futaisi, Anuradha Ganesh [...]

Journal of Child Neurology

Retricted access

Case report

First published 2025-1

Clinical,Pathological,and Molecular Findings in a Mexican Patient With Neuronal Ceroid Lipofuscinosis Type 2: Support for Pathogenicity of the c.1226 G>T Variant and for Presence of Cherry-Red Spot in This Disease

Celso Tomás Corcuera-Delgado, Alfonso Gilberto Ramírez-Ristori, Estela Pérez-Muñoz [...]

Pediatric and Developmental Pathology

Retricted access

Research article

First published 2023-09

Assessment of Safety and Biodistribution of AAVrh.10hCLN2 Following Intracisternal Administration in Nonhuman Primates for the Treatment of CLN2 Batten Disease

Bishnu P. De, Jonathan B. Rosenberg, Nithya Selvan [...]

Human Gene Therapy

Retricted access

Research article

First published 2025-01

Twenty-Year Survival Analysis of Adeno-Associated Virus Vector Serotype 2-Mediated Gene Therapy to the Central Nervous System for CLN2 Disease

Dolan Sondhi, Stephen M. Kaminsky, Jonathan B. Rosenberg [...]

Human Gene Therapy

Retricted access

Research article

First published 2004-11

Administration of a Replication-Deficient Adeno-Associated Virus Gene Transfer Vector Expressing the Human CLN2 cDNA to the Brain of Children with Late Infantile Neuronal Ceroid Lipofuscinosis

Human Gene Therapy

Search results