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Exploring concurrent validity of the CLN2 Clinical Rating Scale: Comparison to PedsQL using cerliponase alfa clinical trial data

PloS one, 2024-05, Vol.19 (5), p.e0302382

2024

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Intramuscular Interferon Beta-1A Therapy Initiated during a First Demyelinating Event in Multiple Sclerosis

The New England journal of medicine, 2000-09, Vol.343 (13), p.898-904

2000

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Long-Term Immunogenicity of Elosulfase Alfa in the Treatment of Morquio A Syndrome: Results From MOR-005, a Phase III Extension Study

Clinical therapeutics, 2017-01, Vol.39 (1), p.118-129.e3

2017

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Clinical Pharmacokinetics and Pharmacodynamics of Cerliponase Alfa, Enzyme Replacement Therapy for CLN2 Disease by Intracerebroventricular Administration

Clinical and translational science, 2021-03, Vol.14 (2), p.635-644

2021

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An Adapted Clinical Measurement Tool for the Key Symptoms of CLN2 Disease

Journal of inborn errors of metabolism and screening, 2018, Vol.6, p.232640981878838

2018

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Study of Intraventricular Cerliponase Alfa for CLN2 Disease

The New England journal of medicine, 2018-05, Vol.378 (20), p.1898-1907

2018

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Cerliponase alfa for the treatment of CLN2 disease in a patient cohort including children under 3 years of age

Molecular genetics and metabolism, 2024-02, Vol.141 (2), p.108034, Article 108034

2024

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Cerliponase alfa for the treatment of CLN2 disease in a patient cohort including children younger than three years: Interim results from an ongoing clinical study

Molecular genetics and metabolism, 2021-02, Vol.132 (2), p.S95-S95

2021

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Efficacy and safety of enzyme replacement therapy with BMN 110 (elosulfase alfa) for Morquio A syndrome (mucopolysaccharidosis IVA): a phase 3 randomised placebo-controlled study

Journal of inherited metabolic disease, 2014-11, Vol.37 (6), p.979-990

2014

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Immunogenicity to cerliponase alfa intracerebroventricular enzyme replacement therapy for CLN2 disease: Results from a Phase 1/2 study

Clinical immunology (Orlando, Fla.), 2018-12, Vol.197, p.68-76

2018

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Safety and efficacy of cerliponase alfa in children with neuronal ceroid lipofuscinosis type 2 (CLN2 disease): an open-label extension study

Lancet neurology, 2024-01, Vol.23 (1), p.60-70

2024

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Persistent treatment effect of cerliponase alfa in children with CLN2 disease: A > 4 year update from an ongoing multicenter extension study

Molecular genetics and metabolism, 2020-02, Vol.129 (2), p.S145-S145

2020

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Cerliponase alfa for the treatment of CLN2 disease in an expanded patient cohort including children younger than three years: Interim results from an ongoing clinical study

Molecular genetics and metabolism, 2020-02, Vol.129 (2), p.S145-S145

2020

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The Morquio A Clinical Assessment Program: Baseline results illustrating progressive, multisystemic clinical impairments in Morquio A subjects

Molecular genetics and metabolism, 2013-05, Vol.109 (1), p.54-61

2013

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Disease characteristics and progression in patients with late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease: an observational cohort study

The lancet child & adolescent health, 2018-08, Vol.2 (8), p.582-590

2018

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Persistent treatment effect of cerliponase alfa in children with CLN2 disease: A 3 year update from an ongoing multicenter extension study

Molecular genetics and metabolism, 2019-02, Vol.126 (2), p.S133-S133

2019

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Long-term endurance and safety of elosulfase alfa enzyme replacement therapy in patients with Morquio A syndrome

Molecular genetics and metabolism, 2016-09, Vol.119 (1-2), p.131-143

2016

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Using the general linear mixed model to analyse unbalanced repeated measures and longitudinal data

Statistics in medicine, 1997-10, Vol.16 (20), p.2349-2380

1997

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Long-term safety and efficacy of intracerebroventricular enzyme replacement therapy with cerliponase alfa in children with CLN2 disease: Two year results from an ongoing multicenter extension study

Molecular genetics and metabolism, 2018-02, Vol.123 (2), p.S126-S127

2018

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Long-term safety and efficacy of intracerebroventricular enzyme replacement therapy with cerliponase alfa in children with CLN2 disease: interim results from an ongoing multicenter, multinational extension study

Molecular genetics and metabolism, 2017-01, Vol.120 (1-2), p.S120-S120

2017

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Longitudinal analysis of endurance and respiratory function from a natural history study of Morquio A syndrome

Molecular genetics and metabolism, 2015-02, Vol.114 (2), p.186-194

2015

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Immunogenicity to cerliponase alfa, an enzyme replacement therapy for patients with CLN2 disease: results from a phase 1/2 study

Molecular genetics and metabolism, 2017-01, Vol.120 (1-2), p.S35-S35

2017

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Impact of long-term elosulfase alfa treatment on pulmonary function in patients with Morquio syndrome type A

Molecular genetics and metabolism, 2016-02, Vol.117 (2), p.S56-S56

2016

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Joint models for efficient estimation in proportional hazards regression models

Statistics in medicine, 2003-07, Vol.22 (13), p.2137-2148

2003

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Does the Sedative Agent Facilitate Emergency Rapid Sequence Intubation?

Academic emergency medicine, 2003-06, Vol.10 (6), p.612-620

2003

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Exploring concurrent validity of the CLN2 Clinical Rating Scale: Comparison to PedsQL using cerliponase alfa clinical trial data

Intramuscular Interferon Beta-1A Therapy Initiated during a First Demyelinating Event in Multiple Sclerosis

Long-Term Immunogenicity of Elosulfase Alfa in the Treatment of Morquio A Syndrome: Results From MOR-005, a Phase III Extension Study

Clinical Pharmacokinetics and Pharmacodynamics of Cerliponase Alfa, Enzyme Replacement Therapy for CLN2 Disease by Intracerebroventricular Administration

An Adapted Clinical Measurement Tool for the Key Symptoms of CLN2 Disease

Study of Intraventricular Cerliponase Alfa for CLN2 Disease

Cerliponase alfa for the treatment of CLN2 disease in a patient cohort including children under 3 years of age

Cerliponase alfa for the treatment of CLN2 disease in a patient cohort including children younger than three years: Interim results from an ongoing clinical study

Efficacy and safety of enzyme replacement therapy with BMN 110 (elosulfase alfa) for Morquio A syndrome (mucopolysaccharidosis IVA): a phase 3 randomised placebo-controlled study

Immunogenicity to cerliponase alfa intracerebroventricular enzyme replacement therapy for CLN2 disease: Results from a Phase 1/2 study

Safety and efficacy of cerliponase alfa in children with neuronal ceroid lipofuscinosis type 2 (CLN2 disease): an open-label extension study

Persistent treatment effect of cerliponase alfa in children with CLN2 disease: A > 4 year update from an ongoing multicenter extension study

Cerliponase alfa for the treatment of CLN2 disease in an expanded patient cohort including children younger than three years: Interim results from an ongoing clinical study

The Morquio A Clinical Assessment Program: Baseline results illustrating progressive, multisystemic clinical impairments in Morquio A subjects

Disease characteristics and progression in patients with late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease: an observational cohort study

Persistent treatment effect of cerliponase alfa in children with CLN2 disease: A 3 year update from an ongoing multicenter extension study

Long-term endurance and safety of elosulfase alfa enzyme replacement therapy in patients with Morquio A syndrome

Using the general linear mixed model to analyse unbalanced repeated measures and longitudinal data

Long-term safety and efficacy of intracerebroventricular enzyme replacement therapy with cerliponase alfa in children with CLN2 disease: Two year results from an ongoing multicenter extension study

Long-term safety and efficacy of intracerebroventricular enzyme replacement therapy with cerliponase alfa in children with CLN2 disease: interim results from an ongoing multicenter, multinational extension study

Longitudinal analysis of endurance and respiratory function from a natural history study of Morquio A syndrome

Immunogenicity to cerliponase alfa, an enzyme replacement therapy for patients with CLN2 disease: results from a phase 1/2 study

Impact of long-term elosulfase alfa treatment on pulmonary function in patients with Morquio syndrome type A

Joint models for efficient estimation in proportional hazards regression models

Does the Sedative Agent Facilitate Emergency Rapid Sequence Intubation?

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