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A Canadian success story: world-first to treat Fabry disease with gene therapy


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IMAGE: Darren Bidulka rests after his modified blood stem cells were transplanted into him at the Foothills Medical Centre in Calgary in 2017, allowing him to stop his enzyme therapy. (From.
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Credit: Courtesy of Darren Bidulka
(Thursday, Feb. 25, 2021, Toronto) Results of a world-first Canadian pilot study on patients treated with gene therapy for Fabry disease show that the treatment is working and safe.
The Canadian research team was the first to use gene therapy in 2017 to treat patients with Fabry disease, a rare, chronic illness that can damage major organs and shorten lives. They report their findings today in the journal ....

City Of , United Kingdom , Stephen Couban , Darren Bidulka , Aneal Khan , Michael West , Armand Keating , Christiane Auray Blais , Ryan Deveau , Anthony Rupar , Amicus Therapeutics , London Health Sciences Centre , University Health Network , Nature Communications , Michener Institute For Education , Alberta Health Services , Takeda Pharmaceuticals , University Of Toronto , Fund Professor , Kidney Foundation Of Canada , Avrobio Inc , Alberta Children Hospital Research Institute , Toronto Rehabilitation Institute , Cumming School Of Medicine , Alberta Health Service Foothills Medical Centre , Senior Scientist At Princess Margaret Cancer Centre ,