ASGCT Releases Patient Education Resources on Fabry Disease

ASGCT Staff - October 07, 2020

We're highlighting the latest resources on our new Patient Education site, this week focusing on Fabry disease.

ASGCT is highlighting its new resources focusing on gene therapy for Fabry disease, which are now available on the new Patient Education site.

Fabry disease is a rare genetic disorder caused by a lacking enzyme due to a faulty gene. Without enough of this key enzyme, a part inside cells called lysosomes are unable to do their job of breaking down waste. Gene therapy aims to offer a one-time treatment to slow or ideally stop progression of the disease by introducing a working GLA gene to provide instructions to the cells to produce the deficient enzyme.

This is the third group of resources from ASGCT's Patient Education Program. During this round, we're releasing new pages, videos, and infographics on the Patient Education site. Next week, we'll highlight Patient Journey, a set of resources to help guide patients through the challenging journey from diagnosis to pursuing gene therapy treatment.

Let us know what you think of the new site and collection of resources by sending feedback to ASGCT Patient Outreach Manager Ali Kujawski.

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