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Last Updated: 10/31/2025

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Found 2642 publications

Correction: Open-label evaluation of oral trehalose in patients with neuronal ceroid lipofuscinoses.

Correction: Open-label evaluation of oral trehalose in patients with neuronal ceroid lipofuscinoses.

Journal: Journal of neurology
Published: October 15, 2025

Bioenergetic Profiling Applied to a Zebrafish Model of Neuronal Ceroid Lipofuscinosis, a Lysosomal Storage Disorder.

Bioenergetic Profiling Applied to a Zebrafish Model of Neuronal Ceroid Lipofuscinosis, a Lysosomal Storage Disorder.

Journal: Methods in molecular biology (Clifton, N.J.)
Published: October 13, 2025

Atlas of Lysosomal Aging Reveals a Molecular Clock of Storage Disorder-Associated Metabolites.

Atlas of Lysosomal Aging Reveals a Molecular Clock of Storage Disorder-Associated Metabolites.

Journal: bioRxiv : the preprint server for biology
Published: October 03, 2025

Visual Recovery and Neurological Stabilization Following Miglustat Treatment in Pediatric CLN3 Disease.

Visual Recovery and Neurological Stabilization Following Miglustat Treatment in Pediatric CLN3 Disease.

Journal: Journal of child neurology
Published: September 18, 2025

Assessment of motor deterioration in a cynomolgus macaque with neuronal ceroid lipofuscinosis type 2 disease using two behavioral analyses.

Assessment of motor deterioration in a cynomolgus macaque with neuronal ceroid lipofuscinosis type 2 disease using two behavioral analyses.

Journal: The Journal of veterinary medical science
Published: September 18, 2025

CLN2 Disease: Current Understandings, Challenges, and Future Directions.

CLN2 Disease: Current Understandings, Challenges, and Future Directions.

Journal: Journal of child neurology
Published: September 18, 2025

Identifying Drug Repurposing Candidates for CLN3 Targeting Proteomics Expression Profile.

Identifying Drug Repurposing Candidates for CLN3 Targeting Proteomics Expression Profile.

Journal: Proceedings. IEEE International Conference on Bioinformatics and Biomedicine
Published: September 17, 2025

Buried Bumper Syndrome With Full Gastric Wall Penetration Managed With Delayed Replacement: A Conservative Approach.

Buried Bumper Syndrome With Full Gastric Wall Penetration Managed With Delayed Replacement: A Conservative Approach.

Journal: Cureus
Published: September 12, 2025

Is Miglustat an Effective Treatment for CLN3 (Batten) Disease?

Is Miglustat an Effective Treatment for CLN3 (Batten) Disease?

Journal: Neurology
Published: September 09, 2025

Long-Term Open-Label Study Evaluating Oral Miglustat Treatment in Patients With Neuronal Ceroid Lipofuscinosis Type 3.

Long-Term Open-Label Study Evaluating Oral Miglustat Treatment in Patients With Neuronal Ceroid Lipofuscinosis Type 3.

Journal: Neurology
Published: September 09, 2025

Discovery of Functionalized 1H-Benzo[d]imidazoles That Confer Protective Effects in a Phenotypic CLN3 Disease Patient-Derived iPSC Model.

Discovery of Functionalized 1H-Benzo[d]imidazoles That Confer Protective Effects in a Phenotypic CLN3 Disease Patient-Derived iPSC Model.

Journal: Journal of medicinal chemistry
Published: September 01, 2025

Age at onset and gene variants predict lifespan and disease duration in childhood neuronal ceroid lipofuscinoses.

Age at onset and gene variants predict lifespan and disease duration in childhood neuronal ceroid lipofuscinoses.

Journal: Developmental medicine and child neurology
Published: August 15, 2025
Showing 1-12 of 2,642

Last Updated: 10/31/2025