Studienbeschreibung

HOSFOS

HOSFOS Registry

A global, multinational, non-interventional, drug registry with longitudinal data collection

A global, multinational, non-interventional, drug registry with longitudinal data collection

16.09.2020

Untersuchte Krankheit, Gesundheitsproblem

• E75.2 - Sonstige Sphingolipidosen

Einschlusskriterien

18 - 99

Alle

Fabry disease Population
Each patient must meet the following criteria to be enrolled in this registry:
1. Patients with a documented diagnosis of Fabry disease, which may include a genetic mutation analysis.
2. Patients who are untreated, currently treated with Replagal, or previously treated with any enzyme replacement therapy (ERT).
3. Signed and dated written informed consent from the patient or, for patients aged <18 years (or as per local regulation), parent and/or patient's legally authorized representative (LAR), and assent of the minor where applicable. If a patient is unable to read or if a legally acceptable representative is unable to read, an impartial witness should be present during the informed consent discussion and should sign and personally date the informed consent.

Ausschlusskriterien

Patients who meet any of the following criteria will be excluded from the Registry.
1. Patients who are receiving treatment with an ERT for Fabry disease other than Replagal.
2. Patients enrolled in a clinical study (except a Replagal open-label clinical study), are not eligible until they have completed or withdrawn from the clinical study.

Charakteristika

Nicht-interventionell

Nicht zutreffend