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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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The humanistic burden of hereditary angioedema: results from the Burden of Illness Study in Europe

Allergy and asthma proceedings, 2014-01, Vol.35 (1), p.47-53

2014

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Socioeconomic burden of hereditary angioedema: results from the hereditary angioedema burden of illness study in Europe

Orphanet journal of rare diseases, 2014-07, Vol.9 (1), p.99-99

2014

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Investigating health-related quality of life in rare diseases: a case study in utility value determination for patients with CLN2 disease (neuronal ceroid lipofuscinosis type 2)

Orphanet journal of rare diseases, 2021-05, Vol.16 (1), p.217-217, Article 217

2021

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Acetylator Status Impacts Amifampridine Phosphate (Firdapse™) Pharmacokinetics and Exposure to a Greater Extent Than Renal Function

Clinical therapeutics, 2017-07, Vol.39 (7), p.1360-1370

2017

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Pregnancy in patients with mucopolysaccharidosis: a case series

Molecular genetics and metabolism reports, 2016-09, Vol.8 (C), p.111-115

2016

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Burden of Illness in Hereditary Angioedema: A Conceptual Model

Acta dermato-venereologica, 2015-07, Vol.95 (6), p.706-710

2015

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Burden of Illness in Hereditary Angioedema: A Conceptual Model

Acta dermato-venereologica, 2015, Vol.95 (6), p.706-710

2015

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Expert Opinions on Managing Fertility and Pregnancy in Patients With Mucopolysaccharidosis

Journal of inborn errors of metabolism and screening, 2016, Vol.4, p.232640981666937

2016

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Enzyme replacement therapy initiated in adulthood: Findings from the mucopolysaccharidosis VI Clinical Surveillance Program

Molecular genetics and metabolism, 2019-08, Vol.127 (4), p.355-360

2019

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Enzyme replacement therapy outcomes across the disease spectrum: Findings from the mucopolysaccharidosis VI Clinical Surveillance Program

Journal of inherited metabolic disease, 2019-05, Vol.42 (3), p.519-526

2019

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Enzyme replacement therapy in patients with mucopolysaccharidosis type VI: Updated findings from the MPS VI clinical surveillance program

Molecular genetics and metabolism, 2019-02, Vol.126 (2), p.S68-S68

2019

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Mucopolysaccharidosis type VI enzyme replacement therapy outcomes across the disease spectrum: findings from the MPS VI clinical surveillance program

Molecular genetics and metabolism, 2018-02, Vol.123 (2), p.S59-S59

2018

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Mucopolysaccharidosis type VI enzyme replacement therapy initiated in adulthood: Findings from the MPS VI clinical surveillance program

Molecular genetics and metabolism, 2018-02, Vol.123 (2), p.S59-S59

2018

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Cervical cord compression in mucopolysaccharidosis VI (MPS VI): Findings from the MPS VI Clinical Surveillance Program (CSP)

Molecular genetics and metabolism, 2016-08, Vol.118 (4), p.310-318

2016

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Time Trade-Off Utilities for Hereditary Angioedema Health and Caregiver States

PharmacoEconomics - open, 2022-03, Vol.6 (2), p.231-239

2022

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Impact of elosulfase alfa in patients with morquio A syndrome who have limited ambulation: An open‐label, phase 2 study

American journal of medical genetics. Part A, 2017-02, Vol.173 (2), p.375-383

2017

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Estimation of EuroQol 5-Dimensions health status utility values in hereditary angioedema

Patient preference and adherence, 2016-01, Vol.10, p.1699-1707

2016

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Impact of elosulfase alfa in patients with Morquio syndrome type A who have limited ambulation: An open-label, phase 2 study

Molecular genetics and metabolism, 2016-02, Vol.117 (2), p.S55-S55

2016

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Pregnancy in individuals with mucopolysaccharidosis (MPS): A case series

Molecular genetics and metabolism, 2016-02, Vol.117 (2), p.S109-S109

2016

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Management of fertility and pregnancy in individuals with mucopolysaccharidosis (MPS)

Molecular genetics and metabolism, 2016-02, Vol.117 (2), p.S109-S110

2016

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Pregnancy in patients with mucopolysaccharidosis: a case series

Molecular genetics and metabolism reports, 2016, Vol.8, p.111-115

2016

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Overview of the current status of familial hypercholesterolaemia care in over 60 countries - The EAS Familial Hypercholesterolaemia Studies Collaboration (FHSC)

Atherosclerosis, 2018-10, Vol.277, p.234-255

2018

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

The humanistic burden of hereditary angioedema: results from the Burden of Illness Study in Europe

Socioeconomic burden of hereditary angioedema: results from the hereditary angioedema burden of illness study in Europe

Investigating health-related quality of life in rare diseases: a case study in utility value determination for patients with CLN2 disease (neuronal ceroid lipofuscinosis type 2)

Acetylator Status Impacts Amifampridine Phosphate (Firdapse™) Pharmacokinetics and Exposure to a Greater Extent Than Renal Function

Pregnancy in patients with mucopolysaccharidosis: a case series

Burden of Illness in Hereditary Angioedema: A Conceptual Model

Burden of Illness in Hereditary Angioedema: A Conceptual Model

Expert Opinions on Managing Fertility and Pregnancy in Patients With Mucopolysaccharidosis

Enzyme replacement therapy initiated in adulthood: Findings from the mucopolysaccharidosis VI Clinical Surveillance Program

Enzyme replacement therapy outcomes across the disease spectrum: Findings from the mucopolysaccharidosis VI Clinical Surveillance Program

Enzyme replacement therapy in patients with mucopolysaccharidosis type VI: Updated findings from the MPS VI clinical surveillance program

Mucopolysaccharidosis type VI enzyme replacement therapy outcomes across the disease spectrum: findings from the MPS VI clinical surveillance program

Mucopolysaccharidosis type VI enzyme replacement therapy initiated in adulthood: Findings from the MPS VI clinical surveillance program

Cervical cord compression in mucopolysaccharidosis VI (MPS VI): Findings from the MPS VI Clinical Surveillance Program (CSP)

Time Trade-Off Utilities for Hereditary Angioedema Health and Caregiver States

Impact of elosulfase alfa in patients with morquio A syndrome who have limited ambulation: An open‐label, phase 2 study

Estimation of EuroQol 5-Dimensions health status utility values in hereditary angioedema

Impact of elosulfase alfa in patients with Morquio syndrome type A who have limited ambulation: An open-label, phase 2 study

Pregnancy in individuals with mucopolysaccharidosis (MPS): A case series

Management of fertility and pregnancy in individuals with mucopolysaccharidosis (MPS)

Pregnancy in patients with mucopolysaccharidosis: a case series

Overview of the current status of familial hypercholesterolaemia care in over 60 countries - The EAS Familial Hypercholesterolaemia Studies Collaboration (FHSC)

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