CLN1 Disease Latest Advances

Translational lipidomics reveals BMP and its precursor LPG as biomarkers for CLN5 Batten disease.

Journal: bioRxiv : the preprint server for biology

Published: March 27, 2026

CLN2 disease: why early diagnosis matters more than ever.

Journal: European journal of paediatric neurology : EJPN : official journal of the European Paediatric Neurology Society

Published: March 13, 2026

Sunken eyes as a peculiar finding in neuronal ceroid lipofuscinoses.

Journal: Arquivos de neuro-psiquiatria

Published: February 27, 2026

Deficient de-S-acylation in aging and CLN1 contributes to lyso-mitochondrial dysfunction, lipid dyshomeostasis, and resultant lipofuscin biogenesis.

Journal: Research square

Published: February 23, 2026

Neurodevelopmental and Psychiatric Disorders and the Use of Psychotropic Medications in a National Sample of Individuals With Juvenile Neuronal Ceroid Lipofuscinosis.

Journal: Journal of child neurology

Published: February 05, 2026

Patient and Family Perspective on Transition from Ventricular Access Device to Chest-Sited Port for Intracerebroventricular Infusion in CLN2 Disease.

Journal: Children (Basel, Switzerland)

Published: February 02, 2026

A Flupirtine Benzyl Carbamate Improves Neurocognitive Deficits and Molecular Pathology in the Cln6nclf Mouse.

Journal: Cells

Published: January 27, 2026

Systemic AAV9 Gene Therapy Mitigates Neuromuscular Junction Degeneration and Muscle Atrophy in a Mouse Model of CLN1 Disease.

Journal: International journal of molecular sciences

Published: January 27, 2026

High-resolution promoter interaction analysis implicates genes involved in the activation of Type 3 Innate Lymphoid Cells in autoimmune disease risk.

Journal: bioRxiv : the preprint server for biology

Published: January 23, 2026

Prodromal pathogenesis of CLN7 Batten Disease revealed by multimodal biomarkers in macaques.

Journal: bioRxiv : the preprint server for biology

Published: January 23, 2026

Distinct lysosomal dysfunction patterns of GRN deficiency in the CNS implicate progranulin in cell type-specific protein sorting.

Journal: bioRxiv : the preprint server for biology

Published: January 09, 2026

Brain-Directed AAV Gene Therapy Rescues a Mouse Model of the CLN5 Form of Neuronal Ceroid Lipofuscinosis Disease and Normalizes a Blood Plasma Biomarker of Neurodegeneration.

Journal: Human gene therapy

Published: December 29, 2025

CLN1 Disease Latest Advances

Find the Latest Research About CLN1 Disease

Translational lipidomics reveals BMP and its precursor LPG as biomarkers for CLN5 Batten disease.

Translational lipidomics reveals BMP and its precursor LPG as biomarkers for CLN5 Batten disease.

CLN2 disease: why early diagnosis matters more than ever.

CLN2 disease: why early diagnosis matters more than ever.

Sunken eyes as a peculiar finding in neuronal ceroid lipofuscinoses.

Sunken eyes as a peculiar finding in neuronal ceroid lipofuscinoses.

Deficient de-S-acylation in aging and CLN1 contributes to lyso-mitochondrial dysfunction, lipid dyshomeostasis, and resultant lipofuscin biogenesis.

Deficient de-S-acylation in aging and CLN1 contributes to lyso-mitochondrial dysfunction, lipid dyshomeostasis, and resultant lipofuscin biogenesis.

Neurodevelopmental and Psychiatric Disorders and the Use of Psychotropic Medications in a National Sample of Individuals With Juvenile Neuronal Ceroid Lipofuscinosis.

Neurodevelopmental and Psychiatric Disorders and the Use of Psychotropic Medications in a National Sample of Individuals With Juvenile Neuronal Ceroid Lipofuscinosis.

Patient and Family Perspective on Transition from Ventricular Access Device to Chest-Sited Port for Intracerebroventricular Infusion in CLN2 Disease.

Patient and Family Perspective on Transition from Ventricular Access Device to Chest-Sited Port for Intracerebroventricular Infusion in CLN2 Disease.

A Flupirtine Benzyl Carbamate Improves Neurocognitive Deficits and Molecular Pathology in the Cln6nclf Mouse.

A Flupirtine Benzyl Carbamate Improves Neurocognitive Deficits and Molecular Pathology in the Cln6nclf Mouse.

Systemic AAV9 Gene Therapy Mitigates Neuromuscular Junction Degeneration and Muscle Atrophy in a Mouse Model of CLN1 Disease.

Systemic AAV9 Gene Therapy Mitigates Neuromuscular Junction Degeneration and Muscle Atrophy in a Mouse Model of CLN1 Disease.

High-resolution promoter interaction analysis implicates genes involved in the activation of Type 3 Innate Lymphoid Cells in autoimmune disease risk.

High-resolution promoter interaction analysis implicates genes involved in the activation of Type 3 Innate Lymphoid Cells in autoimmune disease risk.

Prodromal pathogenesis of CLN7 Batten Disease revealed by multimodal biomarkers in macaques.

Prodromal pathogenesis of CLN7 Batten Disease revealed by multimodal biomarkers in macaques.

Distinct lysosomal dysfunction patterns of GRN deficiency in the CNS implicate progranulin in cell type-specific protein sorting.

Distinct lysosomal dysfunction patterns of GRN deficiency in the CNS implicate progranulin in cell type-specific protein sorting.

Brain-Directed AAV Gene Therapy Rescues a Mouse Model of the CLN5 Form of Neuronal Ceroid Lipofuscinosis Disease and Normalizes a Blood Plasma Biomarker of Neurodegeneration.

Brain-Directed AAV Gene Therapy Rescues a Mouse Model of the CLN5 Form of Neuronal Ceroid Lipofuscinosis Disease and Normalizes a Blood Plasma Biomarker of Neurodegeneration.