New clinical research study for Fabry from Protalix

The BALANCE Study is a clinical research study that will compare the safety and efficacy of an investigational medication (a potential new medication) with an approved medication (agalsidase beta, also known as Fabrazyme) in patients with Fabry disease. The investigational medication is a type of medication called “enzyme replacement therapy,” or ERT. The investigational medication has been designed to help the body break down the particular fat (lipid molecule) that builds up in the body’s cells because of Fabry disease.

The investigational medication in the BALANCE Study is a new ERT. It is designed to last longer in the body, which might help protect the body’s organs from the effects of Fabry disease and reduce symptoms.

Patients with Fabry disease may be able to take part if they:

  • are 18–60 years of age
  • are experiencing symptoms of Fabry disease,
    such as pain or clusters of dark spots on the
    skin known as angiokeratomas
  • have been receiving agalsidase beta infusions
    for approximately 1 year.

For patients interested in taking part, the study team will ask some medical history questions and carry out health assessments at the study center to see if the study is a good fit.

For more information see the BALANCE Study leaflet. Patients can also visit or email

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