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Canada launches first gene therapy trial for Fabry disease
Date:1/24/2013

infusion of medication. Although the infusions help manage his illness, they aren't a cure and they're something he has to organize his life around.

Armstrong, who was diagnosed in 2007, has experienced a thickening in one of the walls of his heart due to Fabry disease. "Even if the research meant that I had to come in once every six months for Enzyme Replacement Therapy, instead of every two weeks, then it would still be another step toward living a normal life," Armstrong says.

In the current pre-clinical phase of the study, researchers hope to demonstrate Armstrong's corrected stem cells are able to provide a source of enzyme in a specially adapted mouse that has Fabry disease. After this is accomplished, the second phase will repeat the experiment using a new donor with all the checks and balances needed for a human trial. Once the pre-clinical experimental results have satisfied the regulatory requirements of Health Canada, the team aims to treat the first human Fabry disease patient, which they estimate will be within two years.


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Contact: Gregory Harris
gregory.harris@albertahealthservices.ca
403-619-3108
Alberta Health Services
Source:Eurekalert

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