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Updated: Jul 4, 2026
10:16
In Vitro Enzyme Measurement to Test Pharmacological Chaperone Responsiveness in Fabry and Pompe Disease
Published on: December 20, 2017
Home-based infusion therapy for patients with Fabry disease
1Charles Dent Metabolic Unit, National Hospital for Neurology and Neurosurgery, London, UK.
British Journal of Nursing (Mark Allen Publishing)
|June 20, 2008
Summary
Home infusion therapy for Fabry disease using agalsidase beta offers a safe and practical alternative to hospital treatments. This patient-centered approach improves quality of life and optimizes hospital resources.
Area of Science:
- Biochemistry
- Genetics
- Pharmacology
Background:
- Fabry disease is a progressive, life-threatening inherited disorder requiring lifelong enzyme replacement therapy.
- Hospital-based agalsidase therapy is often perceived as inconvenient by patients and healthcare providers.
- Home-based infusion therapy presents a patient-preferred alternative for managing Fabry disease.
Purpose of the Study:
- To review clinical experiences with agalsidase beta home infusion therapy.
- To outline the organization of safe, patient-centered homecare for Fabry disease.
- To assess the feasibility and impact of home infusion therapy.
Main Methods:
- Review of collective clinical experiences with agalsidase beta home infusion.
- Identification of prerequisites for safe home therapy (stable patient, suitable environment).
- Discussion of nurse-assisted and self-care infusion models.
Main Results:
- Home infusion therapy is safe and practical, significantly improving patient quality of life.
- Home therapy reduces constraints on hospital resources.
- Eligible patients can benefit from home infusion therapy without delay.
Conclusions:
- Home-based agalsidase beta infusion therapy is a viable and beneficial treatment for Fabry disease.
- Patient-centered homecare models enhance treatment convenience and adherence.
- The principles of home infusion therapy can be applied to other enzyme replacement therapies.
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