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Efficacy of idebenone on respiratory function in patients with Duchenne muscular dystrophy not using glucocorticoids (DELOS): a double-blind randomised placebo-controlled phase 3 trial

The Lancet (British edition), 2015-05, Vol.385 (9979), p.1748-1757

2015

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A randomized placebo-controlled trial of idebenone in Leber's hereditary optic neuropathy

Brain (London, England : 1878), 2011-09, Vol.134 (9), p.2677-2686

2011

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Features of idebenone and related short-chain quinones that rescue ATP levels under conditions of impaired mitochondrial complex I

PloS one, 2012-04, Vol.7 (4), p.e36153-e36153

2012

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Predictors of loss of ambulation in Friedreich's ataxia

EClinicalMedicine, 2020-01, Vol.18, p.100213-100213, Article 100213

2020

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Phenotypic variation of FXN compound heterozygotes in a Friedreich ataxia cohort

Annals of clinical and translational neurology, 2024-05, Vol.11 (5), p.1110-1121

2024

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Idebenone in Friedreich ataxia cardiomyopathy—results from a 6-month phase III study (IONIA)

The American heart journal, 2011-03, Vol.161 (3), p.639-645.e1

2011

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Evaluating mFARS in pediatric Friedreich's ataxia: Insights from the FACHILD study

Annals of clinical and translational neurology, 2024-05, Vol.11 (5), p.1290-1300

2024

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Harmonizing results of ataxia rating scales: mFARS, SARA, and ICARS

Annals of clinical and translational neurology, 2022-12, Vol.9 (12), p.2041-2046

2022

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Propensity matched comparison of omaveloxolone treatment to Friedreich ataxia natural history data

Annals of clinical and translational neurology, 2024-01, Vol.11 (1), p.4-16

2024

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Retinal hypoplasia and degeneration result in vision loss in Friedreich ataxia

Annals of clinical and translational neurology, 2023-08, Vol.10 (8), p.1397-1406

2023

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Digital endpoints for self‐administered home‐based functional assessment in pediatric Friedreich’s ataxia

Annals of clinical and translational neurology, 2021-09, Vol.8 (9), p.1845-1856

2021

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Test–retest reliability of the Friedreich’s ataxia rating scale

Annals of clinical and translational neurology, 2020-09, Vol.7 (9), p.1708-1712

2020

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Scoliosis in Friedreich's ataxia: longitudinal characterization in a large heterogeneous cohort

Annals of clinical and translational neurology, 2021-06, Vol.8 (6), p.1239-1250

2021

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A non-synonymous single nucleotide polymorphism in SIRT6 predicts neurological severity in Friedreich ataxia

Frontiers in molecular biosciences, 2022-09, Vol.9, p.933788-933788

2022

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A Novel Metric for Predicting Severity of Disease Features in Friedreich's Ataxia

Movement disorders, 2023-06, Vol.38 (6), p.970-977

2023

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Revacept, an Inhibitor of Platelet Adhesion in Symptomatic Carotid Stenosis: A Multicenter Randomized Phase II Trial

Stroke (1970), 2022-09, Vol.53 (9), p.STROKEAHA121037006-2729

2022

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Natural History of Friedreich Ataxia: Heterogeneity of Neurologic Progression and Consequences for Clinical Trial Design

Neurology, 2022-10, Vol.99 (14), p.e1499

2022

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Friedreich’s Ataxia related Diabetes: Epidemiology and management practices

Diabetes research and clinical practice, 2022-04, Vol.186, p.109828-109828, Article 109828

2022

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Peroxomolybdenum Complexes as Epoxidation Catalysts in Biphasic Hydrogen Peroxide Activation: Raman Spectroscopic Studies and Density Functional Calculations

Chemistry : a European journal, 1999-11, Vol.5 (11), p.3237-3251

1999

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Characterization of Cardiac-Onset Initial Presentation in Friedreich Ataxia

Pediatric cardiology, 2024-03

2024

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Progression characteristics of the European Friedreich's Ataxia Consortium for Translational Studies (EFACTS): a 4-year cohort study

Lancet neurology, 2021-05, Vol.20 (5), p.362-372

2021

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Clinical trial design for Friedreich ataxia - Where are we now and what do we need?

Expert opinion on orphan drugs, 2018-03, Vol.6 (3), p.219-230

2018

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Homology models of dipeptidyl peptidases 8 and 9 with a focus on loop predictions near the active site

Proteins, structure, function, and bioinformatics, 2007-01, Vol.66 (1), p.160-171

2007

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Standing Balance Conditions and Digital Sway Measures for Clinical Trials of Friedreich's Ataxia

Movement disorders, 2024-06, Vol.39 (6), p.996-1005

2024

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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

Journal of neurology, 2012-02, Vol.259 (2), p.284-291

2012

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Efficacy of idebenone on respiratory function in patients with Duchenne muscular dystrophy not using glucocorticoids (DELOS): a double-blind randomised placebo-controlled phase 3 trial

A randomized placebo-controlled trial of idebenone in Leber's hereditary optic neuropathy

Features of idebenone and related short-chain quinones that rescue ATP levels under conditions of impaired mitochondrial complex I

Predictors of loss of ambulation in Friedreich's ataxia

Phenotypic variation of FXN compound heterozygotes in a Friedreich ataxia cohort

Idebenone in Friedreich ataxia cardiomyopathy—results from a 6-month phase III study (IONIA)

Evaluating mFARS in pediatric Friedreich's ataxia: Insights from the FACHILD study

Harmonizing results of ataxia rating scales: mFARS, SARA, and ICARS

Propensity matched comparison of omaveloxolone treatment to Friedreich ataxia natural history data

Retinal hypoplasia and degeneration result in vision loss in Friedreich ataxia

Digital endpoints for self‐administered home‐based functional assessment in pediatric Friedreich’s ataxia

Test–retest reliability of the Friedreich’s ataxia rating scale

Scoliosis in Friedreich's ataxia: longitudinal characterization in a large heterogeneous cohort

A non-synonymous single nucleotide polymorphism in SIRT6 predicts neurological severity in Friedreich ataxia

A Novel Metric for Predicting Severity of Disease Features in Friedreich's Ataxia

Revacept, an Inhibitor of Platelet Adhesion in Symptomatic Carotid Stenosis: A Multicenter Randomized Phase II Trial

Natural History of Friedreich Ataxia: Heterogeneity of Neurologic Progression and Consequences for Clinical Trial Design

Friedreich’s Ataxia related Diabetes: Epidemiology and management practices

Peroxomolybdenum Complexes as Epoxidation Catalysts in Biphasic Hydrogen Peroxide Activation: Raman Spectroscopic Studies and Density Functional Calculations

Characterization of Cardiac-Onset Initial Presentation in Friedreich Ataxia

Progression characteristics of the European Friedreich's Ataxia Consortium for Translational Studies (EFACTS): a 4-year cohort study

Clinical trial design for Friedreich ataxia - Where are we now and what do we need?

Homology models of dipeptidyl peptidases 8 and 9 with a focus on loop predictions near the active site

Standing Balance Conditions and Digital Sway Measures for Clinical Trials of Friedreich's Ataxia

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

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