Deferiprone in Friedreich ataxia: a 6-month randomized controlled trial. (Q53085750)

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12 March 2020

title

Deferiprone in Friedreich ataxia: a 6-month randomized controlled trial (English)

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Kim-Hanh Le Quan Sang

4

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Franco Taroni

10

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3

Martin B Delatycki

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author name string

Massimo Pandolfo

1

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Javier Arpa

2

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Caterina Mariotti

5

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Irene Sanz-Gallego

7

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Geneieve Tai

8

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Mark A Tarnopolsky

9

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Michael Spino

11

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Fernando Tricta

12

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Arnold Munnich

6

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publication date

30 August 2014

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volume

76

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issue

4

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page(s)

509-521

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Friedreich ataxia: the clinical picture

12 March 2020

cites work

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Prevalence gradients of Friedreich's ataxia and R1b haplotype in Europe co-localize, suggesting a common Palaeolithic origin in the Franco-Cantabrian ice age refuge

21 January 2018

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

21 January 2018

1 reference

Cardiomyopathy of Friedreich ataxia

21 January 2018

1 reference

Mortality in Friedreich ataxia

21 January 2018

1 reference

Diabetes in Friedreich ataxia

21 January 2018

1 reference

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

21 January 2018

1 reference

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

21 January 2018

1 reference

Gene regulation and epigenetics in Friedreich's ataxia

21 January 2018

1 reference

Frataxin is reduced in Friedreich ataxia patients and is associated with mitochondrial membranes

21 January 2018

1 reference

Human frataxin is an allosteric switch that activates the Fe-S cluster biosynthetic complex

21 January 2018

1 reference

Biogenesis of iron-sulfur clusters in mammalian cells: new insights and relevance to human disease

21 January 2018

1 reference

Frataxin is essential for extramitochondrial Fe-S cluster proteins in mammalian tissues

21 January 2018

1 reference

Aconitase and mitochondrial iron–sulphur protein deficiency in Friedreich ataxia

21 January 2018

1 reference

Does oxidative stress contribute to the pathology of Friedreich's ataxia? A radical question

21 January 2018

1 reference

Increased IRP1 activity in Friedreich ataxia

21 January 2018

1 reference

Redistribution of accumulated cell iron: a modality of chelation with therapeutic implications

21 January 2018

1 reference

Cell functions impaired by frataxin deficiency are restored by drug-mediated iron relocation

21 January 2018

1 reference

Selective iron chelation in Friedreich ataxia: biologic and clinical implications

21 January 2018

1 reference

Measuring Friedreich ataxia: Interrater reliability of a neurologic rating scale.

21 January 2018

1 reference

Measuring Friedreich ataxia: complementary features of examination and performance measures

21 January 2018

1 reference

Reporting and interpretation of SF-36 outcomes in randomised trials: systematic review

21 January 2018

1 reference

Friedreich ataxia clinical outcome measures: natural history evaluation in 410 participants

21 January 2018

1 reference

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

21 January 2018

1 reference

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

21 January 2018

1 reference

A review of Friedreich ataxia clinical trial results

21 January 2018

1 reference

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

21 January 2018

1 reference

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

21 January 2018

1 reference

Triple therapy with deferiprone, idebenone and riboflavin in Friedreich's ataxia - open-label trial

21 January 2018

1 reference

Deferiprone targets aconitase: implication for Friedreich's ataxia treatment

21 January 2018

1 reference

Iron-dependent regulation of frataxin expression: implications for treatment of Friedreich ataxia

21 January 2018

1 reference

Pharmacological screening using an FXN-EGFP cellular genomic reporter assay for the therapy of Friedreich ataxia

21 January 2018

1 reference

DNA triplet repeats mediate heterochromatin-protein-1-sensitive variegated gene silencing

21 January 2018

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21 January 2018

Identifiers

DOI

10.1002/ANA.24248

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12 March 2020

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