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Histone deacetylases suppress CGG repeat-induced neurodegeneration via transcriptional silencing in models of fragile X tremor ataxia syndrome

PLoS genetics, 2010-12, Vol.6 (12), p.e1001240

2010

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Designing an exploratory phase 2b platform trial in NASH with correlated, co-primary binary endpoints

PloS one, 2023-03, Vol.18 (3), p.e0281674-e0281674

2023

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Platform trials to overcome major shortcomings of traditional clinical trials in non-alcoholic steatohepatitis? Pros and cons

Journal of hepatology, 2023-02, Vol.78 (2), p.442-447

2023

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

Lancet neurology, 2007-10, Vol.6 (10), p.878-886

2007

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Review article: The need for more efficient and patient‐oriented drug development pathways in NASH—setting the scene for platform trials

Alimentary pharmacology & therapeutics, 2023-05, Vol.57 (9), p.948-961

2023

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Clinical features of spinal and bulbar muscular atrophy

Brain (London, England : 1878), 2009-12, Vol.132 (12), p.3242-3251

2009

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Altered gene expression and DNA damage in peripheral blood cells from Friedreich's ataxia patients: cellular model of pathology

PLoS genetics, 2010-01, Vol.6 (1), p.e1000812

2010

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Effects of rifampin, cyclosporine A, and probenecid on the pharmacokinetic profile of canagliflozin, a sodium glucose co-transporter 2 inhibitor, in healthy participants

International journal of clinical pharmacology and therapeutics, 2015-02, Vol.53 (2), p.115-128

2015

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Impaired mitochondrial function in psychiatric disorders

Nature reviews. Neuroscience, 2012-05, Vol.13 (5), p.293-307

2012

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Mechanism of Increased LDL (Low-Density Lipoprotein) and Decreased Triglycerides With SGLT2 (Sodium-Glucose Cotransporter 2) Inhibition

Arteriosclerosis, thrombosis, and vascular biology, 2018-09, Vol.38 (9), p.2207-2216

2018

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Expression of expanded polyglutamine targets profilin for degradation and alters actin dynamics

Neurobiology of disease, 2008-06, Vol.30 (3), p.365-374

2008

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Standard and modified statistical mune evaluations in spinal-bulbar muscular atrophy

Muscle & nerve, 2009-11, Vol.40 (5), p.809-814

2009

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Biochemical and Phenotypic Abnormalities in Kynurenine Aminotransferase II-Deficient Mice

Molecular and Cellular Biology, 2004-08, Vol.24 (16), p.6919-6930

2004

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Exercise Capacity and Idebenone Intervention in Children and Adolescents With Friedreich Ataxia

Archives of physical medicine and rehabilitation, 2010-07, Vol.91 (7), p.1044-1050

2010

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Efficacy and safety of glucagon‐like peptide‐1/glucagon receptor co‐agonist JNJ‐64565111 in individuals with type 2 diabetes mellitus and obesity: A randomized dose‐ranging study

Clinical obesity, 2021-04, Vol.11 (2), p.n/a

2021

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Trichostatin A increases SMN expression and survival in a mouse model of spinal muscular atrophy

The Journal of clinical investigation, 2007-03, Vol.117 (3), p.659-671

2007

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Pharmacokinetics and pharmacodynamics of canagliflozin in pediatric patients with type 2 diabetes

Pediatric diabetes, 2018-06, Vol.19 (4), p.649-655

2018

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Assessing Function and Endurance in Adults with Spinal and Bulbar Muscular Atrophy: Validity of the Adult Myopathy Assessment Tool

Rehabilitation Research and Practice, 2014-01, Vol.2014 (2014), p.1-16

2014

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The Subclinical Cardiomyopathy of Friedreich's Ataxia in a Pediatric Population

Journal of cardiac failure, 2018-10, Vol.24 (10), p.672-679

2018

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Therapeutics development for triplet repeat expansion diseases

Nature reviews. Genetics, 2005-10, Vol.6 (10), p.756-767

2005

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Clinical experience with high-dose idebenone in Friedreich ataxia

Journal of neurology, 2009-03, Vol.256 (Suppl 1), p.42-45

2009

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

Brain (London, England : 1878), 2013, Vol.136 (Pt 1), p.259-268

2013

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Effect of food on the pharmacokinetics of canagliflozin, a sodium glucose co-transporter 2 inhibitor, and assessment of dose proportionality in healthy participants

Clinical pharmacology in drug development, 2015-07, Vol.4 (4), p.279-286

2015

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Predictors of progression in patients with Friedreich ataxia

Movement disorders, 2008-10, Vol.23 (14), p.2026-2032

2008

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Histone deacetylases suppress CGG repeat-induced neurodegeneration via transcriptional silencing in models of fragile X tremor ataxia syndrome

Designing an exploratory phase 2b platform trial in NASH with correlated, co-primary binary endpoints

Platform trials to overcome major shortcomings of traditional clinical trials in non-alcoholic steatohepatitis? Pros and cons

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

Review article: The need for more efficient and patient‐oriented drug development pathways in NASH—setting the scene for platform trials

Clinical features of spinal and bulbar muscular atrophy

Altered gene expression and DNA damage in peripheral blood cells from Friedreich's ataxia patients: cellular model of pathology

Effects of rifampin, cyclosporine A, and probenecid on the pharmacokinetic profile of canagliflozin, a sodium glucose co-transporter 2 inhibitor, in healthy participants

Impaired mitochondrial function in psychiatric disorders

Mechanism of Increased LDL (Low-Density Lipoprotein) and Decreased Triglycerides With SGLT2 (Sodium-Glucose Cotransporter 2) Inhibition

Expression of expanded polyglutamine targets profilin for degradation and alters actin dynamics

Standard and modified statistical mune evaluations in spinal-bulbar muscular atrophy

Biochemical and Phenotypic Abnormalities in Kynurenine Aminotransferase II-Deficient Mice

Exercise Capacity and Idebenone Intervention in Children and Adolescents With Friedreich Ataxia

Efficacy and safety of glucagon‐like peptide‐1/glucagon receptor co‐agonist JNJ‐64565111 in individuals with type 2 diabetes mellitus and obesity: A randomized dose‐ranging study

Trichostatin A increases SMN expression and survival in a mouse model of spinal muscular atrophy

Pharmacokinetics and pharmacodynamics of canagliflozin in pediatric patients with type 2 diabetes

Assessing Function and Endurance in Adults with Spinal and Bulbar Muscular Atrophy: Validity of the Adult Myopathy Assessment Tool

The Subclinical Cardiomyopathy of Friedreich's Ataxia in a Pediatric Population

Therapeutics development for triplet repeat expansion diseases

Efficacy and safety of dutasteride in patients with spinal and bulbar muscular atrophy: a randomised placebo-controlled trial

Clinical experience with high-dose idebenone in Friedreich ataxia

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

Effect of food on the pharmacokinetics of canagliflozin, a sodium glucose co-transporter 2 inhibitor, and assessment of dose proportionality in healthy participants

Predictors of progression in patients with Friedreich ataxia

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