Search results: Sage Journals

Restricted accessReview articleFirst published January, 2026

CLN2 Disease: Current Understandings,Challenges,and Future Directions

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

Journal of Child Neurology

Open accessResearch articleFirst 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 accessResearch articleFirst published May, 2021

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 accessResearch articleFirst published July, 2021

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 accessArticle commentaryFirst published December, 2023

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

Gordon F. Buchanan

Epilepsy Currents

Restricted accessOtherFirst published May, 2007

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

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

Journal of Child Neurology

Restricted accessCase reportFirst published January, 2025

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

Restricted accessResearch articleFirst published September, 2023

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

Restricted accessResearch articleFirst published January, 2025

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

Restricted accessResearch articleFirst published November, 2004

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

Restricted accessReview articleFirst published January, 2026

CLN2 Disease: Current Understandings,Challenges,and Future Directions

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

Journal of Child Neurology

Open accessResearch articleFirst 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 accessResearch articleFirst published May, 2021

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 accessResearch articleFirst published July, 2021

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 accessArticle commentaryFirst published December, 2023

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

Gordon F. Buchanan

Epilepsy Currents

Restricted accessOtherFirst published May, 2007

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

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

Journal of Child Neurology

Restricted accessCase reportFirst published January, 2025

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

Restricted accessResearch articleFirst published September, 2023

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

Restricted accessResearch articleFirst published January, 2025

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

Restricted accessResearch articleFirst published November, 2004

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