Safety and potential efficacy of gemfibrozil as a supportive treatment for children with late infantile neuronal ceroid lipofuscinosis and other lipid storage disorders

Moving towards effective therapeutic strategies for Neuronal Ceroid Lipofuscinosis

(2016) Ryan D. Geraets et al.   Orphanet Journal of Rare Diseases

Activation of Sterol Regulatory Element Binding Factors by Fenofibrate and Gemfibrozil Stimulates Myelination in Zebrafish

(2016) Yoshifumi Ashikawa et al.   Frontiers in Pharmacology

Multifocal retinopathy in Dachshunds with CLN2 neuronal ceroid lipofuscinosis

(2015) Rebecca E.H. Whiting et al.   EXPERIMENTAL EYE RESEARCH

Fibrates inhibit the apoptosis of Batten disease lymphoblast cells via autophagy recovery and regulation of mitochondrial membrane potential

(2015) Minho Hong et al.   IN VITRO CELLULAR & DEVELOPMENTAL BIOLOGY-ANIMAL

Activation of Peroxisome Proliferator-activated Receptor α Induces Lysosomal Biogenesis in Brain Cells

(2015) Arunava Ghosh et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Nonclinical evaluation of CNS-administered TPP1 enzyme replacement in canine CLN2 neuronal ceroid lipofuscinosis

(2015) Brian R. Vuillemenot et al.   MOLECULAR GENETICS AND METABOLISM

AAV gene transfer delays disease onset in a TPP1-deficient canine model of the late infantile form of Batten disease

(2015) Martin L. Katz et al.   Science Translational Medicine

Enzyme replacement therapy attenuates disease progression in a canine model of late-infantile neuronal ceroid lipofuscinosis (CLN2 disease)

(2014) Martin L. Katz et al.   JOURNAL OF NEUROSCIENCE RESEARCH

Oral cysteamine bitartrate and N-acetylcysteine for patients with infantile neuronal ceroid lipofuscinosis: a pilot study

(2014) Sondra W Levin et al.   LANCET NEUROLOGY

TFEB regulates lysosomal proteostasis

(2013) Wensi Song et al.   HUMAN MOLECULAR GENETICS

Human iPSC models of neuronal ceroid lipofuscinosis capture distinct effects of TPP1 and CLN3 mutations on the endocytic pathway

(2013) Xenia Lojewski et al.   HUMAN MOLECULAR GENETICS

Batten Disease

(2013) Sarah-Bianca Dolisca et al.   JOURNAL OF CHILD NEUROLOGY

Chronic oral administration of minocycline to sheep with ovine CLN6 neuronal ceroid lipofuscinosis maintains pharmacological concentrations in the brain but does not suppress neuroinflammation or disease progression

(2013) Graham W Kay et al.   Journal of Neuroinflammation

Challenges in the Pharmacologic Management of Obesity and Secondary Dyslipidemia in Children and Adolescents

(2013) Mary Jayne Kennedy et al.   PEDIATRIC DRUGS

Gemfibrozil, a Lipid-lowering Drug, Increases Myelin Genes in Human Oligodendrocytes via Peroxisome Proliferator-activated Receptor-β

(2012) Malabendu Jana et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Intravenous high-dose enzyme replacement therapy with recombinant palmitoyl–protein thioesterase reduces visceral lysosomal storage and modestly prolongs survival in a preclinical mouse model of infantile neuronal ceroid lipofuscinosis

(2012) Jie Hu et al.   MOLECULAR GENETICS AND METABOLISM

Gemfibrozil, a Lipid Lowering Drug, Inhibits the Activation of Primary Human Microglia Via Peroxisome Proliferator-Activated Receptor β

(2012) Malabendu Jana et al.   NEUROCHEMICAL RESEARCH

New nomenclature and classification scheme for the neuronal ceroid lipofuscinoses

(2012) R. E. Williams et al.   NEUROLOGY

Long-Term Expression and Safety of Administration of AAVrh.10hCLN2 to the Brain of Rats and Nonhuman Primates for the Treatment of Late Infantile Neuronal Ceroid Lipofuscinosis

(2012) Dolan Sondhi et al.   Human Gene Therapy Methods

Identification and Characterization of Pharmacological Chaperones to Correct Enzyme Deficiencies in Lysosomal Storage Disorders

(2011) Kenneth J. Valenzano et al.   ASSAY AND DRUG DEVELOPMENT TECHNOLOGIES

Transcriptional Activation of Lysosomal Exocytosis Promotes Cellular Clearance

(2011) Diego L. Medina et al.   DEVELOPMENTAL CELL

Characterization of the CLEAR network reveals an integrated control of cellular clearance pathways

(2011) Michela Palmieri et al.   HUMAN MOLECULAR GENETICS

Update of the mutation spectrum and clinical correlations of over 360 mutations in eight genes that underlie the neuronal ceroid lipofuscinoses

(2011) Maria Kousi et al.   HUMAN MUTATION

Large-volume Intrathecal Enzyme Delivery Increases Survival of a Mouse Model of Late Infantile Neuronal Ceroid Lipofuscinosis

(2011) Su Xu et al.   MOLECULAR THERAPY

Palmitoyl:protein thioesterase (PPT1) inhibitors can act as pharmacological chaperones in infantile Batten disease

(2010) Glyn Dawson et al.   BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS

Current therapies for the soluble lysosomal forms of neuronal ceroid lipofuscinosis

(2010) Andrew M.S. Wong et al.   BIOCHEMICAL SOCIETY TRANSACTIONS

Immunosuppression alters disease severity in juvenile Batten disease mice

(2010) Sabrina S. Seehafer et al.   JOURNAL OF NEUROIMMUNOLOGY

Temporary inhibition of AMPA receptors induces a prolonged improvement of motor performance in a mouse model of juvenile Batten disease

(2010) Attila D. Kovács et al.   NEUROPHARMACOLOGY

Neuroprotection of Host Cells by Human Central Nervous System Stem Cells in a Mouse Model of Infantile Neuronal Ceroid Lipofuscinosis

(2009) Stanley J. Tamaki et al.   Cell Stem Cell

Gemfibrozil, stretching arms beyond lipid lowering

(2009) Avik Roy et al.   IMMUNOPHARMACOLOGY AND IMMUNOTOXICOLOGY

Human recombinant palmitoyl-protein thioesterase-1 (PPT1) for preclinical evaluation of enzyme replacement therapy for infantile neuronal ceroid lipofuscinosis

(2009) Jui-Yun Lu et al.   MOLECULAR GENETICS AND METABOLISM

Survival advantage of neonatal CNS gene transfer for late infantile neuronal ceroid lipofuscinosis

(2008) Dolan Sondhi et al.   EXPERIMENTAL NEUROLOGY

Treatment of Late Infantile Neuronal Ceroid Lipofuscinosis by CNS Administration of a Serotype 2 Adeno-Associated Virus Expressing CLN2 cDNA

(2008) Stefan Worgall et al.   HUMAN GENE THERAPY

A pilot study in the efficacy and safety of gemfibrozil in a pediatric population

(2008) Courtney M. Smalley et al.   Journal of Clinical Lipidology

Intraventricular Enzyme Replacement Improves Disease Phenotypes in a Mouse Model of Late Infantile Neuronal Ceroid Lipofuscinosis

(2008) Michael Chang et al.   MOLECULAR THERAPY

Attenuation of AMPA receptor activity improves motor skills in a mouse model of juvenile Batten disease

(2007) Attila D. Kovács et al.   EXPERIMENTAL NEUROLOGY