How much time does fabry treatment really take? new study measures the burden
NCT ID NCT04281537
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This completed study looked at how much time healthcare professionals spend preparing and giving enzyme replacement therapy (ERT) to adults with Fabry disease. It also measured the impact on patients and their caregivers, including time, costs, and quality of life. The goal was to describe the real-world experience of ERT treatment across multiple countries.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
agalsidase alfa and agalsidase beta (enzyme replacement therapy)
What this could lead to
If successful, this study could help clinics plan better and reduce the burden of treatment for Fabry disease patients and their caregivers.
What could go wrong
This is an observational study, not a treatment trial. It describes current practices but does not test a new therapy or change outcomes.
Disclaimer
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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.
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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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
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