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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. ....

Kathryn Downey , Sarah Halprin , Aneal Khan , Rare Disease Day , Ehllers Danlos Syndrome , Finallyi Ve , Assured Income , Severely Handicapped ,

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. ....

United States , Kelly Newell , Emilita Dela Cruz , Aneal Khan , Christine White , Elisabeth Linton , Canadian Organization For Rare Disorders , Foundation Of Canada , Association Des Patients Immunod , Takeda Pharmaceutical Company Limited , Takeda Canada Inc , National Gaucher Foundation Of Canada , Canadian Fabry Association , Sickle Cell Disease Association Of Canada , National Strategy For Drugs , Takeda Canada , Sanfilippo Syndrome , Kerstyn Lane , Hereditary Angioedema Type , Vice President , Regional Director , National Strategy , Rare Diseases , National Gaucher Foundation , Rute Fernandes , General Manager ,

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 ....

United States , Terry Pirovolakis , Manohar Shroff , Darren Bidulka , Aneal Khan , Shuhui Sun , Michael Pirovolakis , Jim Dowling , David Malkin , Cancer Genetics Program , Drug Administration , Precision Child Health , Institute For Stem Cell , Child Health Research At Sickkids , Postmedia Network Inc , Division Of Neurology , Regeneration Of The Chinese Academy Sciences , Foothills Medical Centre , Health Canada , Children Foundation , Sick Children , Foundation Chair , Child Health Research , Genome Biology , Stem Cell , Chinese Academy ,

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

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