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Efficacy and Safety of Abatacept in Lupus Nephritis: A Twelve‐Month, Randomized, Double‐Blind Study

Arthritis & rheumatology (Hoboken, N.J.), 2014-02, Vol.66 (2), p.379-389

2014

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Cardiovascular outcomes in Fabry disease are linked to severity of chronic kidney disease

Heart (British Cardiac Society), 2015-02, Vol.101 (4), p.287-293

2015

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GLA-modified RNA treatment lowers GB3 levels in iPSC-derived cardiomyocytes from Fabry-affected individuals

American journal of human genetics, 2023-09, Vol.110 (9), p.1600-1605

2023

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Safety and efficacy of pegunigalsidase alfa in patients with Fabry disease who were previously treated with agalsidase alfa: results from BRIDGE, a phase 3 open-label study

Orphanet journal of rare diseases, 2023-10, Vol.18 (1), p.1-332, Article 332

2023

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Agalsidase Alfa and Kidney Dysfunction in Fabry Disease

Journal of the American Society of Nephrology, 2009-05, Vol.20 (5), p.1132-1139

2009

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The Effectiveness of Long-Term Agalsidase Alfa Therapy in the Treatment of Fabry Nephropathy

Clinical journal of the American Society of Nephrology, 2012-01, Vol.7 (1), p.60-69

2012

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Oral Migalastat HCl Leads to Greater Systemic Exposure and Tissue Levels of Active α-Galactosidase A in Fabry Patients when Co-Administered with Infused Agalsidase

PloS one, 2015-08, Vol.10 (8), p.e0134341-e0134341

2015

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Safety and pharmacodynamic effects of a pharmacological chaperone on α-galactosidase A activity and globotriaosylceramide clearance in Fabry disease: report from two phase 2 clinical studies

Orphanet journal of rare diseases, 2012-11, Vol.7 (1), p.91-91

2012

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Effects of enzyme replacement therapy in Fabry disease—A comprehensive review of the medical literature

Genetics in medicine, 2010-11, Vol.12 (11), p.668-679

2010

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Long-term follow-up of renal function in patients treated with migalastat for Fabry disease

Molecular genetics and metabolism reports, 2021-09, Vol.28, p.100786-100786, Article 100786

2021

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Migalastat HCl reduces globotriaosylsphingosine (lyso-Gb3) in Fabry transgenic mice and in the plasma of Fabry patients

PloS one, 2013-03, Vol.8 (3), p.e57631-e57631

2013

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Corrigendum to “Long-term follow-up of renal function in patients treated with migalastat for Fabry disease” [Bichet et al., MGM Reports; 28 (2021) 100786]

Molecular genetics and metabolism reports, 2021-12, Vol.29, p.100807-100807, Article 100807

2021

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Elevated Lyso-Gb3 Suggests the R118C GLA Mutation Is a Pathological Fabry Variant

JIMD Reports, Volume 45, 2019, Vol.45, p.95-98

2019

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Antiepileptic Medications Increase Osteoporosis Risk in Male Fabry Patients: Bone Mineral Density in an Australian Cohort

JIMD Reports, Volume 17, 2014-01, Vol.17, p.29-36

2014

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Canagliflozin and Cardiovascular and Renal Outcomes in Type 2 Diabetes Mellitus and Chronic Kidney Disease in Primary and Secondary Cardiovascular Prevention Groups: Results From the Randomized CREDENCE Trial

Circulation (New York, N.Y.), 2019-08, Vol.140 (9), p.739-750

2019

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Effect of Oral Methylprednisolone on Clinical Outcomes in Patients With IgA Nephropathy: The TESTING Randomized Clinical Trial

JAMA : the journal of the American Medical Association, 2017-08, Vol.318 (5), p.432

2017

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The N215S Fabry disease mutation is not just a “cardiac” variant: Strong evidence of renal involvement

Molecular genetics and metabolism, 2018-02, Vol.123 (2), p.S138-S138

2018

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Long-term outcomes in patients with Fabry disease who were treated with agalsidase alfa for more than nineteen years: The Fabry Outcome Survey

Molecular genetics and metabolism, 2023-02, Vol.138 (2), p.107118, Article 107118

2023

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Pegunigalsidase alfa, a novel PEGylated enzyme replacement therapy for Fabry disease, provides sustained plasma concentrations and favorable pharmacodynamics: A 1‐year Phase 1/2 clinical trial

Journal of inherited metabolic disease, 2019-05, Vol.42 (3), p.534-544

2019

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Efficacy of the pharmacologic chaperone migalastat in a subset of male patients with the classic phenotype of Fabry disease and migalastat-amenable variants: data from the phase 3 randomized, multicenter, double-blind clinical trial and extension study

Genetics in medicine, 2019-09, Vol.21 (9), p.1987-1997

2019

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Screening, diagnosis, and management of patients with Fabry disease: conclusions from a “Kidney Disease: Improving Global Outcomes” (KDIGO) Controversies Conference

Kidney international, 2017-02, Vol.91 (2), p.284-293

2017

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Cardiovascular Events in Patients With Fabry Disease

Journal of the American College of Cardiology, 2011-03, Vol.57 (9), p.1093-1099

2011

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A simple method for quantification of plasma globotriaosylsphingosine: Utility for Fabry disease

Molecular genetics and metabolism, 2017-09, Vol.122 (1-2), p.121-125

2017

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Mycophenolate versus Azathioprine as Maintenance Therapy for Lupus Nephritis

The New England journal of medicine, 2011-11, Vol.365 (20), p.1886-1895

2011

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Mycophenolate Mofetil versus Cyclophosphamide for Induction Treatment of Lupus Nephritis

Journal of the American Society of Nephrology, 2009-05, Vol.20 (5), p.1103-1112

2009

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Efficacy and Safety of Abatacept in Lupus Nephritis: A Twelve‐Month, Randomized, Double‐Blind Study

Cardiovascular outcomes in Fabry disease are linked to severity of chronic kidney disease

GLA-modified RNA treatment lowers GB3 levels in iPSC-derived cardiomyocytes from Fabry-affected individuals

Safety and efficacy of pegunigalsidase alfa in patients with Fabry disease who were previously treated with agalsidase alfa: results from BRIDGE, a phase 3 open-label study

Agalsidase Alfa and Kidney Dysfunction in Fabry Disease

The Effectiveness of Long-Term Agalsidase Alfa Therapy in the Treatment of Fabry Nephropathy

Oral Migalastat HCl Leads to Greater Systemic Exposure and Tissue Levels of Active α-Galactosidase A in Fabry Patients when Co-Administered with Infused Agalsidase

Safety and pharmacodynamic effects of a pharmacological chaperone on α-galactosidase A activity and globotriaosylceramide clearance in Fabry disease: report from two phase 2 clinical studies

Effects of enzyme replacement therapy in Fabry disease—A comprehensive review of the medical literature

Long-term follow-up of renal function in patients treated with migalastat for Fabry disease

Migalastat HCl reduces globotriaosylsphingosine (lyso-Gb3) in Fabry transgenic mice and in the plasma of Fabry patients

Corrigendum to “Long-term follow-up of renal function in patients treated with migalastat for Fabry disease” [Bichet et al., MGM Reports; 28 (2021) 100786]

Elevated Lyso-Gb3 Suggests the R118C GLA Mutation Is a Pathological Fabry Variant

Antiepileptic Medications Increase Osteoporosis Risk in Male Fabry Patients: Bone Mineral Density in an Australian Cohort

Canagliflozin and Cardiovascular and Renal Outcomes in Type 2 Diabetes Mellitus and Chronic Kidney Disease in Primary and Secondary Cardiovascular Prevention Groups: Results From the Randomized CREDENCE Trial

Effect of Oral Methylprednisolone on Clinical Outcomes in Patients With IgA Nephropathy: The TESTING Randomized Clinical Trial

The N215S Fabry disease mutation is not just a “cardiac” variant: Strong evidence of renal involvement

Long-term outcomes in patients with Fabry disease who were treated with agalsidase alfa for more than nineteen years: The Fabry Outcome Survey

Pegunigalsidase alfa, a novel PEGylated enzyme replacement therapy for Fabry disease, provides sustained plasma concentrations and favorable pharmacodynamics: A 1‐year Phase 1/2 clinical trial

Efficacy of the pharmacologic chaperone migalastat in a subset of male patients with the classic phenotype of Fabry disease and migalastat-amenable variants: data from the phase 3 randomized, multicenter, double-blind clinical trial and extension study

Screening, diagnosis, and management of patients with Fabry disease: conclusions from a “Kidney Disease: Improving Global Outcomes” (KDIGO) Controversies Conference

Cardiovascular Events in Patients With Fabry Disease

A simple method for quantification of plasma globotriaosylsphingosine: Utility for Fabry disease

Mycophenolate versus Azathioprine as Maintenance Therapy for Lupus Nephritis

Mycophenolate Mofetil versus Cyclophosphamide for Induction Treatment of Lupus Nephritis

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