Safety and potential efficacy of gemfibrozil as a supportive treatment for children with late infantile neuronal ceroid lipofuscinosis and other lipid storage disorders (Q33808609)

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

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scholarly article

1 reference

Europe PubMed Central

29 July 2017

9 references

24 March 2019

22 April 2019

25 April 2019

5 September 2019

9 September 2019

7 November 2019

8 November 2019

19 November 2019

15 August 2020

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

1 reference

Europe PubMed Central

29 July 2017

Infantile neuronal ceroid lipofuscinosis

0 references

0 references

juvenile neuronal ceroid lipofuscinosis

3 references

24 March 2019

22 April 2019

25 April 2019

1 reference

5 September 2019

neuronal ceroid lipofuscinosis

6 references

5 September 2019

9 September 2019

7 November 2019

8 November 2019

19 November 2019

15 August 2020

1 reference

9 September 2019

carboxylic acid

1 reference

7 November 2019

1 reference

8 November 2019

1 reference

7 November 2019

1 reference

19 November 2019

Jansky–Bielschowsky disease

1 reference

based on heuristic

inferred from title

author name string

Kyeongsoon Kim

1

1 reference

Europe PubMed Central

29 July 2017

Hynda K Kleinman

2

1 reference

Europe PubMed Central

29 July 2017

Hahn-Jun Lee

3

1 reference

Europe PubMed Central

29 July 2017

Kalipada Pahan

4

1 reference

Europe PubMed Central

29 July 2017

language of work or name

1 reference

based on heuristic

inferred from title

publication date

17 June 2017

10 references

Europe PubMed Central

29 July 2017

24 March 2019

22 April 2019

25 April 2019

5 September 2019

9 September 2019

7 November 2019

8 November 2019

19 November 2019

15 August 2020

Orphanet Journal of Rare Diseases

10 references

Europe PubMed Central

29 July 2017

24 March 2019

22 April 2019

25 April 2019

5 September 2019

9 September 2019

7 November 2019

8 November 2019

19 November 2019

15 August 2020

12

1 reference

Europe PubMed Central

29 July 2017

113

1 reference

Europe PubMed Central

29 July 2017

1

1 reference

Europe PubMed Central

29 July 2017

copyright license

Creative Commons Attribution 4.0 International

9 references

Europe PubMed Central

24 March 2019

Europe PubMed Central

22 April 2019

Europe PubMed Central

25 April 2019

Europe PubMed Central

5 September 2019

Europe PubMed Central

9 September 2019

Europe PubMed Central

7 November 2019

Europe PubMed Central

8 November 2019

Europe PubMed Central

19 November 2019

Europe PubMed Central

15 August 2020

copyright status

0 references

on focus list of Wikimedia project

0 references

https://scigraph.springernature.com/pub.10.1186/s13023-017-0663-8

0 references

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5474050

4 July 2018

Moving towards effective therapeutic strategies for Neuronal Ceroid Lipofuscinosis

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5474050

4 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5474050

4 July 2018

Genetics of the neuronal ceroid lipofuscinoses (Batten disease).

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5474050

4 July 2018

Bullous dermatosis of childhood induced by gemfibrozil

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5474050

4 July 2018

Cell biology of the NCL proteins: What they do and don't do.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5474050

4 July 2018

Experimental therapies in the neuronal ceroid lipofuscinoses

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5474050

4 July 2018

Future perspectives: Moving towards NCL treatments

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5474050

4 July 2018

Activation of peroxisome proliferator-activated receptor α induces lysosomal biogenesis in brain cells: implications for lysosomal storage disorders

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5474050

4 July 2018

Multifocal retinopathy in Dachshunds with CLN2 neuronal ceroid lipofuscinosis

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5474050

4 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5474050

4 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5474050

4 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5474050

4 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5474050

4 July 2018

Batten disease: clinical aspects, molecular mechanisms, translational science, and future directions

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5474050

4 July 2018

Challenges in the pharmacologic management of obesity and secondary dyslipidemia in children and adolescents

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5474050

4 July 2018

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.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5474050

4 July 2018

Gemfibrozil and fenofibrate, Food and Drug Administration-approved lipid-lowering drugs, up-regulate tripeptidyl-peptidase 1 in brain cells via peroxisome proliferator-activated receptor α: implications for late infantile Batten disease therapy

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5474050

4 July 2018

Gemfibrozil, a lipid-lowering drug, increases myelin genes in human oligodendrocytes via peroxisome proliferator-activated receptor-β.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5474050

4 July 2018

New nomenclature and classification scheme for the neuronal ceroid lipofuscinoses

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5474050

4 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5474050

4 July 2018

Gemfibrozil, a lipid lowering drug, inhibits the activation of primary human microglia via peroxisome proliferator-activated receptor β.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5474050

4 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5474050

4 July 2018

Transcriptional activation of lysosomal exocytosis promotes cellular clearance

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5474050

4 July 2018

Large-volume intrathecal enzyme delivery increases survival of a mouse model of late infantile neuronal ceroid lipofuscinosis

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5474050

4 July 2018

Identification and characterization of pharmacological chaperones to correct enzyme deficiencies in lysosomal storage disorders

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5474050

4 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5474050

4 July 2018

Two siblings with familial chylomicronemia syndrome: disease course and effectiveness of early treatment

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5474050

4 July 2018

Current therapies for the soluble lysosomal forms of neuronal ceroid lipofuscinosis

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5474050

4 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5474050

4 July 2018

Immunosuppression alters disease severity in juvenile Batten disease mice

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5474050

4 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5474050

4 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5474050

4 July 2018

Gemfibrozil, stretching arms beyond lipid lowering

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5474050

4 July 2018

A gene network regulating lysosomal biogenesis and function

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5474050

4 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5474050

4 July 2018

Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5474050

4 July 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5474050

4 July 2018

Progress towards understanding disease mechanisms in small vertebrate models of neuronal ceroid lipofuscinosis.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5474050

4 July 2018

High resolution 1H NMR-based metabolomics indicates a neurotransmitter cycling deficit in cerebral tissue from a mouse model of Batten disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5474050

4 July 2018

Stability of lentiviral vector-mediated transgene expression in the brain in the presence of systemic antivector immune responses

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5474050

4 July 2018

Hematopoietic stem cell transplantation in infantile neuronal ceroid lipofuscinosis

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5474050

4 July 2018

The hypolipidaemic effect of gemfibrozil (CI-719) in laboratory animals

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5474050

4 July 2018

Intraventricular enzyme replacement improves disease phenotypes in a mouse model of late infantile neuronal ceroid lipofuscinosis

1 reference

https://api.crossref.org/works/10.1186%2FS13023-017-0663-8

21 January 2018

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

1 reference

https://api.crossref.org/works/10.1186%2FS13023-017-0663-8

21 January 2018

Bone marrow transplantation in late infantile Batten disease and juvenile Batten disease.

1 reference

https://api.crossref.org/works/10.1186%2FS13023-017-0663-8

21 January 2018

Neuroprotection of host cells by human central nervous system stem cells in a mouse model of infantile neuronal ceroid lipofuscinosis

1 reference

https://api.crossref.org/works/10.1186%2FS13023-017-0663-8

21 January 2018

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

1 reference

https://api.crossref.org/works/10.1186%2FS13023-017-0663-8

21 January 2018

Gemfibrozil, food and drug administration-approved lipid-lowering drug, increases longevity in mouse model of late infantile neuronal ceroid lipofuscinosis.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5474050

30 October 2018

The neuronal ceroid-lipofuscinoses: a historical introduction.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5474050

30 October 2018

Gemfibrozil in late preterm and term neonates with moderate jaundice: a randomized controlled trial.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5474050

30 October 2018

The effects of gemfibrozil on hyperlipidemia in children with persistent nephrotic syndrome

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5474050

30 October 2018

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

1 reference

https://pubmed.ncbi.nlm.nih.gov/28623936

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Effect of gemfibrozil on centrifugal behavior of rat peroxisomes and activities of peroxisomal enzymes involved in lipid metabolism

1 reference

https://pubmed.ncbi.nlm.nih.gov/28623936

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

TFEB regulates lysosomal proteostasis

1 reference

https://pubmed.ncbi.nlm.nih.gov/28623936

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Identifiers

10.1186/S13023-017-0663-8

1 reference

Europe PubMed Central

29 July 2017

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0 references

1 reference

Europe PubMed Central

29 July 2017

1 reference

Europe PubMed Central

29 July 2017

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