Understanding Fabry patients and their interest in future therapies

Updated: Mar 4

Rare Disease Research Partners have developed a survey to understand patients with Fabry who are not currently receiving treatment (e.g. Replagal® (agalsidase alfa), Fabrazyme® (agalsidase beta) or Galafold® (migalastat)), and their interest in future therapies.

This information will help to understand the reasons why patients with classical Fabry are not receiving treatment and their perspective on future therapies. This will assist with advocating for the development and access to new treatments with the goal of improving the lives of Fabry individuals and their families around the world.

The survey is open to male individuals with a diagnosis of classical Fabry disease. Participants should be aged 18 years or over.

The online survey should take less than 15 minutes to complete.

No personal identifiers will be collected in this survey and your answers will remain anonymous. The information you provide will be shared with the pharmaceutical company funding this survey.

We are the only registered charity providing professional support to individuals and families affected by MPS, Fabry or a related disease in the UK.

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