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Jay A. Barth

Amicus Therapeutics

in patient
Fabry disease
Medicine
outcome measures
open label

2

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10

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0

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FABry Disease Patient-Reported Outcome-GastroIntestinal (FABPRO-GI): A new Fabry disease-specific gastrointestinal outcomes instrument.

2021 Quality of Life Research
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Long-term efficacy and safety of migalastat treatment in Fabry disease: 30-month results from the open-label extension of the randomized, phase 3 ATTRACT study

2020 Molecular Genetics and Metabolism
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Citations (9)

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