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Novel mobility test to assess functional vision in patients with inherited retinal dystrophies

Clinical & experimental ophthalmology, 2018-04, Vol.46 (3), p.247-259

2018

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A multicenter, retrospective medical record review of X‐linked myotubular myopathy: The recensus study

Muscle & nerve, 2018-04, Vol.57 (4), p.550-560

2018

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Endovascular stent grafting versus open surgical repair of descending thoracic aortic aneurysms in low-risk patients: A multicenter comparative trial

The Journal of thoracic and cardiovascular surgery, 2007-02, Vol.133 (2), p.369-377.e4

2007

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Frequent source plasma donors are not at risk of iron depletion: the Ferritin Levels in Plasma Donor (FLIPD) study

Transfusion (Philadelphia, Pa.), 2018-04, Vol.58 (4), p.951-959

2018

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Enzyme replacement therapy for mucopolysaccharidosis VI: A phase 3, randomized, double-blind, placebo-controlled, multinational study of recombinant human N-acetylgalactosamine 4-sulfatase (recombinant human arylsulfatase B or rhASB) and follow-on, open-label extension study

The Journal of pediatrics, 2006-04, Vol.148 (4), p.533-539.e6

2006

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Efficacy, Safety, and Durability of Voretigene Neparvovec-rzyl in RPE65 Mutation-Associated Inherited Retinal Dystrophy: Results of Phase 1 and 3 Trials

Ophthalmology (Rochester, Minn.), 2019-09, Vol.126 (9), p.1273-1285

2019

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Long-term follow-up of endurance and safety outcomes during enzyme replacement therapy for mucopolysaccharidosis VI: Final results of three clinical studies of recombinant human N-acetylgalactosamine 4-sulfatase

Molecular genetics and metabolism, 2008-08, Vol.94 (4), p.469-475

2008

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Direct comparison of measures of endurance, mobility, and joint function during enzyme-replacement therapy of mucopolysaccharidosis VI (Maroteaux-Lamy syndrome): results after 48 weeks in a phase 2 open-label clinical study of recombinant human N-acetylgalactosamine 4-sulfatase

Pediatrics (Evanston), 2005-06, Vol.115 (6), p.e681

2005

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Durability of Voretigene Neparvovec for Biallelic RPE65-Mediated Inherited Retinal Disease: Phase 3 Results at 3 and 4 Years

Ophthalmology (Rochester, Minn.), 2021-10, Vol.128 (10), p.1460-1468

2021

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Functionalized Copolymers of Isobutylene with Vinyl Phenol: Synthesis, Characterization, and Property

Chinese journal of polymer science, 2019-09, Vol.37 (9), p.919-929

2019

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Enzyme replacement therapy for mucopolysaccharidosis VI: evaluation of long-term pulmonary function in patients treated with recombinant human N-acetylgalactosamine 4-sulfatase

Journal of inherited metabolic disease, 2010-02, Vol.33 (1), p.51-60

2010

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Long‐term survival of participants in the CENTAUR trial of sodium phenylbutyrate‐taurursodiol in amyotrophic lateral sclerosis

Muscle & nerve, 2021-01, Vol.63 (1), p.31-39

2021

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The impact of kidney function on outcomes following high risk myocardial infarction: findings from 27 610 patients

European journal of heart failure, 2014-03, Vol.16 (3), p.289-299

2014

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Cognitive endpoints for therapy development for neuronopathic mucopolysaccharidoses: Results of a consensus procedure

Molecular genetics and metabolism, 2017-06, Vol.121 (2), p.70-79

2017

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Simulation Study of Quantitative Risk Assessment for Bivariate Continuous Outcomes

Risk analysis, 2008-10, Vol.28 (5), p.1415-1430

2008

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Quantitative Risk Assessment for Multivariate Continuous Outcomes with Application to Neurotoxicology: The Bivariate Case

Biometrics, 2005-09, Vol.61 (3), p.757-766

2005

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Results of phase III clinical trial subretinal gene therapy for RPE65-mediated Leber congenital amaurosis (LCA)

Journal of AAPOS, 2016-08, Vol.20 (4), p.e4-e4

2016

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N-Methylformamide in advanced squamous cancer of the uterine cervix : An Eastern Cooperative Oncology Group phase II trial

Investigational new drugs, 2001-01, Vol.19 (3), p.233-237

2001

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Durability of Voretigene Neparvovec for Biallelic RPE65-Mediated Inherited Retinal Disease

Ophthalmology (Rochester, Minn.), 2021-10, Vol.128 (10), p.1460-1468

2021

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How long does gene therapy last? 4-year follow-up of phase 3 voretigene neparvovec trial in RPE65-associated LCA/inherited retinal disease

Journal of AAPOS, 2019-08, Vol.23 (4), p.e7-e7

2019

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Year 3 results and age-stratified analyses for a phase 3 trial of voretigene neparvovec in RPE65 mutation-associated inherited retinal disease

Journal of AAPOS, 2018-08, Vol.22 (4), p.e13-e13

2018

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Design and Analysis of Randomized Clinical Trials

Design and Analysis of Experiments, 2011, Vol.810, p.165-211

2011

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Characterization and wear behavior of WC-0.8Co coating on cast steel rolls by electro-spark deposition

International journal of minerals, metallurgy and materials, 2009-12, Vol.16 (6), p.707-713

2009

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Comparative Study between Robotic Total Thyroidectomy with Central Lymph Node Dissection via Bilateral Axillo-breast Approach and Conventional Open Procedure for Papillary Thyroid Microcarcinoma

Chinese medical journal, 2016-09, Vol.129 (18), p.2160-2166

2016

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Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial

Novel mobility test to assess functional vision in patients with inherited retinal dystrophies

A multicenter, retrospective medical record review of X‐linked myotubular myopathy: The recensus study

Endovascular stent grafting versus open surgical repair of descending thoracic aortic aneurysms in low-risk patients: A multicenter comparative trial

Frequent source plasma donors are not at risk of iron depletion: the Ferritin Levels in Plasma Donor (FLIPD) study

Enzyme replacement therapy for mucopolysaccharidosis VI: A phase 3, randomized, double-blind, placebo-controlled, multinational study of recombinant human N-acetylgalactosamine 4-sulfatase (recombinant human arylsulfatase B or rhASB) and follow-on, open-label extension study

Efficacy, Safety, and Durability of Voretigene Neparvovec-rzyl in RPE65 Mutation-Associated Inherited Retinal Dystrophy: Results of Phase 1 and 3 Trials

Long-term follow-up of endurance and safety outcomes during enzyme replacement therapy for mucopolysaccharidosis VI: Final results of three clinical studies of recombinant human N-acetylgalactosamine 4-sulfatase

Direct comparison of measures of endurance, mobility, and joint function during enzyme-replacement therapy of mucopolysaccharidosis VI (Maroteaux-Lamy syndrome): results after 48 weeks in a phase 2 open-label clinical study of recombinant human N-acetylgalactosamine 4-sulfatase

Durability of Voretigene Neparvovec for Biallelic RPE65-Mediated Inherited Retinal Disease: Phase 3 Results at 3 and 4 Years

Functionalized Copolymers of Isobutylene with Vinyl Phenol: Synthesis, Characterization, and Property

Enzyme replacement therapy for mucopolysaccharidosis VI: evaluation of long-term pulmonary function in patients treated with recombinant human N-acetylgalactosamine 4-sulfatase

Long‐term survival of participants in the CENTAUR trial of sodium phenylbutyrate‐taurursodiol in amyotrophic lateral sclerosis

The impact of kidney function on outcomes following high risk myocardial infarction: findings from 27 610 patients

Cognitive endpoints for therapy development for neuronopathic mucopolysaccharidoses: Results of a consensus procedure

Simulation Study of Quantitative Risk Assessment for Bivariate Continuous Outcomes

Quantitative Risk Assessment for Multivariate Continuous Outcomes with Application to Neurotoxicology: The Bivariate Case

Results of phase III clinical trial subretinal gene therapy for RPE65-mediated Leber congenital amaurosis (LCA)

N-Methylformamide in advanced squamous cancer of the uterine cervix : An Eastern Cooperative Oncology Group phase II trial

Durability of Voretigene Neparvovec for Biallelic RPE65-Mediated Inherited Retinal Disease

How long does gene therapy last? 4-year follow-up of phase 3 voretigene neparvovec trial in RPE65-associated LCA/inherited retinal disease

Year 3 results and age-stratified analyses for a phase 3 trial of voretigene neparvovec in RPE65 mutation-associated inherited retinal disease

Design and Analysis of Randomized Clinical Trials

Characterization and wear behavior of WC-0.8Co coating on cast steel rolls by electro-spark deposition

Comparative Study between Robotic Total Thyroidectomy with Central Lymph Node Dissection via Bilateral Axillo-breast Approach and Conventional Open Procedure for Papillary Thyroid Microcarcinoma

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