Rare Disease Report

Several Treatments in Development for Fabry Disease

MARCH 29, 2015

Fabry disease is an X-linked lysosomal storage disorder that leads to excessive deposition of globotriaosylceramide ( GL-3) throughout the body. Skin, eye, kidney, heart, brain, and peripheral nervous system are highly vulnerable.

There is currently one orphan drug approved for patients with Fabry (Fabrazyme) but there are also many in development. In this exclusive interview, Jack Johnson of  FSIG (Fabry Support and Information Group) talks about the many drugs being studied to improve the treatment options of people with Fabry disease.

For more information about FSIG, visit www.fabry.org

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