Fabry Disease Information

About Fabry Disease

Fabry disease is a lysosomal storage disorder (LSD) that interferes with the body’s ability to break down a specific fatty substance (globotriaosylceramide or Gb3) which accumulates within the body due to deficiency of a specific enzyme (α-galactosidase A). Fabry disease affects both males and females and can present with a number of signs or symptoms of variable degree.

Learn More About Fabry Disease & Shire HGT

If you are a physician looking for more information about Shire HGT's treatment protocol for Fabry disease, please contact OnePathSM by calling 1-866-888-0660 or contact us online. OnePathSM will direct inquiries to appropriate clinical trial management personnel.

Fabry Disease Resources

BraveCommunity.com

BraveCommunity.com is a dynamic source of news, information, and personal insights about certain rare diseases.