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Sara M. Hawley

BioMarin Pharmaceutical

13

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229

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

2018 Molecular Genetics and Metabolism
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Presenting signs and symptoms of MPS: Results of a systematic literature analysis

2018 Molecular Genetics and Metabolism
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Safety, efficacy, and immunogenicity of elosulfase alfa in patients with Morquio syndrome type A participating in 2 sequential open-label studies (MOR-002/MOR-100), representing 5 years of treatment

2018 Molecular Genetics and Metabolism
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Mucopolysaccharidosis type VI enzyme replacement therapy outcomes across the disease spectrum: findings from the MPS VI clinical surveillance program

2018 Molecular Genetics and Metabolism
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Impact of long-term elosulfase alfa on activities of daily living in patients with Morquio A syndrome in an open-label, multi-center, phase 3 extension study

2017 Molecular Genetics and Metabolism
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Clinical outcomes in a subpopulation of adults with Morquio A syndrome: results from a long-term extension study of elosulfase alfa

2017 Orphanet Journal of Rare Diseases
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Diagnosing mucopolysaccharidosis IVA

2013 Journal of Inherited Metabolic Disease
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Clinical overview and treatment options for non-skeletal manifestations of mucopolysaccharidosis type IVA

2013 Journal of Inherited Metabolic Disease
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Expert recommendations for the laboratory diagnosis of MPS VI.

2012 Molecular Genetics and Metabolism
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