Therapeutic approaches for the treatment of Friedreich's ataxia (Q39158402)

9 September 2017

title

Therapeutic approaches for the treatment of Friedreich's ataxia (English)

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9 September 2017

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Cassandra J Strawser

1

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9 September 2017

Kimberly A Schadt

series ordinal

2

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9 September 2017

David R Lynch

series ordinal

3

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9 September 2017

language of work or name

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18 July 2014

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Expert Review of Neurotherapeutics: a key contribution to decision making in the treatment of neurologic and neuropsychiatric disorders

9 September 2017

published in

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9 September 2017

volume

14

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9 September 2017

issue

8

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9 September 2017

page(s)

949-957

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Friedreich ataxia: effects of genetic understanding on clinical evaluation and therapy

9 September 2017

cites work

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https://api.crossref.org/works/10.1586%2F14737175.2014.939173

Clinical and genetic abnormalities in patients with Friedreich's ataxia

21 January 2018

1 reference

https://api.crossref.org/works/10.1586%2F14737175.2014.939173

Mortality in Friedreich ataxia

21 January 2018

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https://api.crossref.org/works/10.1586%2F14737175.2014.939173

Friedreich ataxia: executive control is related to disease onset and GAA repeat length.

21 January 2018

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https://api.crossref.org/works/10.1586%2F14737175.2014.939173

Clinical features of Friedreich's ataxia: classical and atypical phenotypes.

21 January 2018

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https://api.crossref.org/works/10.1586%2F14737175.2014.939173

Friedreich's ataxia: autosomal recessive disease caused by an intronic GAA triplet repeat expansion

21 January 2018

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https://api.crossref.org/works/10.1586%2F14737175.2014.939173

Molecular pathogenesis of Friedreich ataxia

21 January 2018

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https://api.crossref.org/works/10.1586%2F14737175.2014.939173

Rating disease progression of Friedreich's ataxia by the International Cooperative Ataxia Rating Scale: analysis of a 603-patient database.

21 January 2018

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https://api.crossref.org/works/10.1586%2F14737175.2014.939173

Mitochondrial pathophysiology in Friedreich's ataxia

21 January 2018

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https://api.crossref.org/works/10.1586%2F14737175.2014.939173

Idebenone and reduced cardiac hypertrophy in Friedreich's ataxia

21 January 2018

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https://api.crossref.org/works/10.1586%2F14737175.2014.939173

Idebenone treatment in Friedreich patients: one-year-long randomized placebo-controlled trial

21 January 2018

1 reference

https://api.crossref.org/works/10.1586%2F14737175.2014.939173

Idebenone treatment in Friedreich's ataxia: neurological, cardiac, and biochemical monitoring

21 January 2018

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https://api.crossref.org/works/10.1586%2F14737175.2014.939173

Friedreich's ataxia: idebenone treatment in early stage patients

21 January 2018

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https://api.crossref.org/works/10.1586%2F14737175.2014.939173

Idebenone treatment in paediatric and adult patients with Friedreich ataxia: long-term follow-up

21 January 2018

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https://api.crossref.org/works/10.1586%2F14737175.2014.939173

Neurological effects of high-dose idebenone in patients with Friedreich's ataxia: a randomised, placebo-controlled trial

21 January 2018

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https://api.crossref.org/works/10.1586%2F14737175.2014.939173

A phase 3, double-blind, placebo-controlled trial of idebenone in friedreich ataxia

21 January 2018

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https://api.crossref.org/works/10.1586%2F14737175.2014.939173

Assessment of neurological efficacy of idebenone in pediatric patients with Friedreich's ataxia: data from a 6-month controlled study followed by a 12-month open-label extension study

21 January 2018

1 reference

https://api.crossref.org/works/10.1586%2F14737175.2014.939173

Antioxidant use in Friedreich ataxia

21 January 2018

1 reference

https://api.crossref.org/works/10.1586%2F14737175.2014.939173

Triple therapy with darbepoetin alfa, idebenone, and riboflavin in Friedreich's ataxia: an open-label trial

21 January 2018

1 reference

https://api.crossref.org/works/10.1586%2F14737175.2014.939173

Co-enzyme Q10 and idebenone use in Friedreich's ataxia

21 January 2018

1 reference

https://api.crossref.org/works/10.1586%2F14737175.2014.939173

Antioxidant treatment improves in vivo cardiac and skeletal muscle bioenergetics in patients with Friedreich's ataxia

21 January 2018

1 reference

https://api.crossref.org/works/10.1586%2F14737175.2014.939173

Friedreich's Ataxia: disease mechanisms, antioxidant and Coenzyme Q10 therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1586%2F14737175.2014.939173

Antioxidant treatment of patients with Friedreich ataxia: four-year follow-up

21 January 2018

1 reference

https://api.crossref.org/works/10.1586%2F14737175.2014.939173

Coenzyme Q10 and vitamin E deficiency in Friedreich's ataxia: predictor of efficacy of vitamin E and coenzyme Q10 therapy.

21 January 2018

1 reference

https://api.crossref.org/works/10.1586%2F14737175.2014.939173

EPI-743 reverses the progression of the pediatric mitochondrial disease--genetically defined Leigh Syndrome.

21 January 2018

1 reference

https://api.crossref.org/works/10.1586%2F14737175.2014.939173

Effect of EPI-743 on the clinical course of the mitochondrial disease Leber hereditary optic neuropathy

21 January 2018

1 reference

https://api.crossref.org/works/10.1586%2F14737175.2014.939173

Initial experience in the treatment of inherited mitochondrial disease with EPI-743.

21 January 2018

1 reference

https://api.crossref.org/works/10.1586%2F14737175.2014.939173

Development of indole-3-propionic acid (OXIGON) for Alzheimer's disease.

21 January 2018

1 reference

https://api.crossref.org/works/10.1586%2F14737175.2014.939173

Indole-3-propionic acid attenuates neuronal damage and oxidative stress in the ischemic hippocampus

21 January 2018

1 reference

https://api.crossref.org/works/10.1586%2F14737175.2014.939173

Potent neuroprotective properties against the Alzheimer beta-amyloid by an endogenous melatonin-related indole structure, indole-3-propionic acid

21 January 2018

1 reference

https://api.crossref.org/works/10.1586%2F14737175.2014.939173

Deferiprone for the treatment of Friedreich's ataxia.

21 January 2018

1 reference

https://api.crossref.org/works/10.1586%2F14737175.2014.939173

Selective iron chelation in Friedreich ataxia: biologic and clinical implications.

21 January 2018

1 reference

https://api.crossref.org/works/10.1586%2F14737175.2014.939173

Combined therapy with idebenone and deferiprone in patients with Friedreich's ataxia.

21 January 2018

1 reference

https://api.crossref.org/works/10.1586%2F14737175.2014.939173

Functional genomic analysis of frataxin deficiency reveals tissue-specific alterations and identifies the PPARgamma pathway as a therapeutic target in Friedreich's ataxia

21 January 2018

1 reference

https://api.crossref.org/works/10.1586%2F14737175.2014.939173

PGC-1alpha down-regulation affects the antioxidant response in Friedreich's ataxia

21 January 2018

1 reference

https://api.crossref.org/works/10.1586%2F14737175.2014.939173

PARIS (ZNF746) repression of PGC-1α contributes to neurodegeneration in Parkinson's disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1586%2F14737175.2014.939173

Insights into the role of oxidative stress in the pathology of Friedreich ataxia using peroxidation resistant polyunsaturated fatty acids

21 January 2018

1 reference

https://api.crossref.org/works/10.1586%2F14737175.2014.939173

Isotopic reinforcement of essential polyunsaturated fatty acids diminishes nigrostriatal degeneration in a mouse model of Parkinson's disease.

21 January 2018

1 reference

https://api.crossref.org/works/10.1586%2F14737175.2014.939173

Unanswered questions in Friedreich ataxia

21 January 2018

1 reference

https://api.crossref.org/works/10.1586%2F14737175.2014.939173

Recombinant human erythropoietin: effects on frataxin expression in vitro

21 January 2018

1 reference

https://api.crossref.org/works/10.1586%2F14737175.2014.939173

Neurological effects of recombinant human erythropoietin in Friedreich's ataxia: a clinical pilot trial

21 January 2018

1 reference

https://api.crossref.org/works/10.1586%2F14737175.2014.939173

Effects of Erythropoietin on Frataxin Levels and Mitochondrial Function in Friedreich Ataxia – a Dose–Response Trial

21 January 2018

1 reference

https://api.crossref.org/works/10.1586%2F14737175.2014.939173

A TAT-frataxin fusion protein increases lifespan and cardiac function in a conditional Friedreich's ataxia mouse model

21 January 2018

1 reference

https://api.crossref.org/works/10.1586%2F14737175.2014.939173

Interferon gamma upregulates frataxin and corrects the functional deficits in a Friedreich ataxia model.

21 January 2018

1 reference

https://api.crossref.org/works/10.1586%2F14737175.2014.939173

HDAC inhibitors correct frataxin deficiency in a Friedreich ataxia mouse model

21 January 2018

1 reference

https://api.crossref.org/works/10.1586%2F14737175.2014.939173

Rationale for the development of 2-aminobenzamide histone deacetylase inhibitors as therapeutics for Friedreich ataxia

21 January 2018

1 reference

https://api.crossref.org/works/10.1586%2F14737175.2014.939173

Histone deacetylase inhibitors reverse gene silencing in Friedreich's ataxia.

21 January 2018

1 reference

https://api.crossref.org/works/10.1586%2F14737175.2014.939173

Heterochromatinization induced by GAA-repeat hyperexpansion in Friedreich's ataxia can be reduced upon HDAC inhibition by vitamin B3.

21 January 2018

1 reference

https://api.crossref.org/works/10.1586%2F14737175.2014.939173

Prevention and reversal of severe mitochondrial cardiomyopathy by gene therapy in a mouse model of Friedreich's ataxia

21 January 2018

1 reference

https://api.crossref.org/works/10.1586%2F14737175.2014.939173

Infectious Delivery and Expression of a 135 kb Human FRDA Genomic DNA Locus Complements Friedreich's Ataxia Deficiency in Human Cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1586%2F14737175.2014.939173

Prospects for the use of artificial chromosomes and minichromosome-like episomes in gene therapy.

21 January 2018

1 reference

https://api.crossref.org/works/10.1586%2F14737175.2014.939173

Infectious delivery and long-term persistence of transgene expression in the brain by a 135-kb iBAC-FXN genomic DNA expression vector.

21 January 2018

1 reference

https://api.crossref.org/works/10.1586%2F14737175.2014.939173

Functional recovery in a Friedreich's ataxia mouse model by frataxin gene transfer using an HSV-1 amplicon vector

21 January 2018

1 reference

https://api.crossref.org/works/10.1586%2F14737175.2014.939173

Erythropoietin in Friedreich ataxia: No effect on frataxin in a randomized controlled trial

21 January 2018

1 reference

https://api.crossref.org/works/10.1586%2F14737175.2014.939173

10.1586/14737175.2014.939173

21 January 2018

Identifiers

DOI

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9 September 2017

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