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Canadian pilot study uses gene therapy to successfully treat patients with Fabry disease 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 Nature Communications. Being one of the first people in the world to receive this treatment, and seeing how much better I felt afterward, it definitely gives me hope that this can help many other Fabry patients and potentially those with other single gene mutation disorders, says Ryan Deveau, one of the participating patients in Dartmouth, Nova Scotia. Now that I don t have to get the replacement therapy every two weeks, I have more time to spend with my family.

Stephen coubanDarren bidulkaAneal khanRyan deveauMichael westEmily hendersonUniversity health networkNature communicationsAlberta health service foothills medical centreCumming school of medicineNova scotia healthNational institutes of healthAlberta health servicesCollege of wisconsinPrincess margaret cancer centreFund professor