Hidden costs of rare disease treatment revealed in global study
NCT ID NCT04281537
Summary
This study aimed to understand the full burden of receiving enzyme replacement therapy for Fabry disease, a rare genetic disorder. Researchers observed 82 adult patients and their caregivers across multiple countries to measure the time doctors spent on infusions, the time and travel costs for patients and families, and how treatment affects work and quality of life. The goal was to gather real-world data on the challenges of managing this lifelong treatment.
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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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