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Sheela Sitaraman

Amicus Therapeutics

30

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Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL): an international, randomised, double-blind, parallel-group, phase 3 trial

2021 Lancet Neurology
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Use of the patient-reported outcomes measurement information system (PROMIS®) to assess late-onset Pompe disease severity.

2020 Journal of Patient-Reported Outcomes
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PRECLINICAL APPROACHES AND EARLY CLINICAL RESULTS

2019 Neuromuscular Disorders
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Preliminary patient-reported outcomes and safety of advanced and targeted acid α-glucosidase (AT-GAA (ATB200/AT2221) in patients with Pompe disease from the ATB200-02 trial

2019 Molecular Genetics and Metabolism
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Safety and efficacy of advanced and targeted acid α-glucosidase (AT-GAA) (ATB200/AT2221) in ERT-switch nonambulatory patients with Pompe disease: preliminary results from the ATB200-02 trial

2019 Molecular Genetics and Metabolism
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The patient and clinician point of view: living with late-onset Pompe disease

2018 Molecular Genetics and Metabolism
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First-in-human preliminary pharmacokinetic data on a novel recombinant acid α-glucosidase, ATB200, co-administered with the pharmacological chaperone, AT2221, in patients with late-onset Pompe disease

2018 Molecular Genetics and Metabolism
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First-in-human Study of ATB200/AT2221 in Patients with Pompe Disease: Preliminary Results From the ATB200-02 Trial (S22.003)

2018 Neurology
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Duvoglustat HCl Increases Systemic and Tissue Exposure of Active Acid α-Glucosidase in Pompe Patients Co-administered with Alglucosidase α

2017 Molecular Therapy
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Oral Migalastat HCl Leads to Greater Systemic Exposure and Tissue Levels of Active α-Galactosidase A in Fabry Patients when Co-Administered with Infused Agalsidase

2015 PLOS ONE
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