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Rare Disease Day sheds light on prevalence of rare diseases in Canada

Advocates and affected patients are working to raise awareness of the prevalence of rare diseases among Canadians.

New Campaign Celebrates Canadian Rare Disease Changemakers

/CNW/ - Today, 13 Canadian rare disease organizations, in partnership with Takeda Canada Inc. ("Takeda",) unite to launch I Am Number 12, to raise awareness of.

Canadian family raised $3.5M to develop individualized gene therapy for son's rare condition

The family is cautiously optimistic about the improvements they are seeing in Michael, 4, since the gene therapy three months ago

Maladie de Fabry : trois patients arrêtent leur traitement grâce à la thérapie génique

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

 E-Mail 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. view more  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