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William Lang

BioMarin Pharmaceutical

5

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Long-term developmental progression in infants and young children taking sapropterin for phenylketonuria: A two-year analysis of safety and efficacy

2015 Genetics in Medicine
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A randomized, placebo-controlled, double-blind study of sapropterin to treat ADHD symptoms and executive function impairment in children and adults with sapropterin-responsive phenylketonuria

2015 Molecular Genetics and Metabolism
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Preliminary clinical efficacy and safety of BMN 701, GILT-tagged recombinant human acid alpha glucosidase (rhGAA), in late-onset Pompe disease: results of an extension study

2014 Molecular Genetics and Metabolism
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POM-001 phase 1/2 study of BMN 701, GILT-tagged recombinant human (rh) GAA in late-onset Pompe disease: Initial experience in 22 patients

2013 Molecular Genetics and Metabolism
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POM-001 Phase 1/2 Study of BMN 701, GILT-tagged Recombinant Human (rh) GAA in Late-Onset Pompe Disease: Preliminary Report

2012 Molecular Genetics and Metabolism
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