Fabry treatment study reveals hidden time and cost burdens on patients and caregivers
NCT ID NCT04281537
First seen Feb 05, 2026 · Last updated Jun 15, 2026 · Updated 19 times
Summary
This study looked at how enzyme replacement therapy (ERT) for Fabry disease affects the daily lives of patients, their caregivers, and healthcare providers. Researchers measured the time spent preparing and giving the treatment, as well as its impact on patient quality of life, fatigue, and work productivity. The goal was to better understand the real-world experience of managing this rare disease.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Locations
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Amicus Therapeutics, Inc.
Philadelphia, Pennsylvania, 19104, United States
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Dokuz Eylul University Medical Faculty
Izmir, Turkey (Türkiye)
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Ege University Hospital
Izmir, Turkey (Türkiye)
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Gazi University Hospital
Ankara, Turkey (Türkiye)
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Instituto de Genética e Erros Inatos do Metabolismo (IGEIM)
São Paulo, Brazil
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Keio University Hospital
Tokyo, Japan
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National Taiwan University
Taipei, Taiwan
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Taipei Veterans General Hospital
Taipei, Taiwan
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Yokohama Municipal Citizen's Hospital
Yokohama, Japan
Conditions
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