Fabry Disease…The purpose of this study is to observe the safety of agalsidase alfa in Canadian patients with Fabry disease. Principal Investigator Sandra SirrsArea Vancouver
Canadian Fabry Disease Initiative (CFDI) Enzyme Replacement Therapy (ERT) StudyCFDI STUDY with ENZYME REPLACEMENT THERAPY (ERT) Principal Investigator Sandra SirrsBody Locations and Systems BrainArea VancouverAge 5-85
APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated AmyloidosisThe purpose of this study is to evaluate the safety and efficacy of patisiran (ALN-TTR02) in patients with transthyretin (TTR) mediated amyloidosis Principal Investigator Michelle M. MezeiBody Locations and Systems Neurologic DiseasesArea VancouverAge 18-85
A Prospective Observational Study of Patients With Primary Mitochondrial Disease (SPIMM-300)This is an observational study of patients with Primary Mitochondrial Disease with either signs or symptoms suggestive of myopathy. Principal Investigator Anna LehmanBody Locations and Systems Brain and NervesArea VancouverAge 16-65 years
Completed Study A8241021…This study is a 12 month open label extension study of PF-02545920 20 mg dosed BID following study A8241021 in subjects with HD. Primary endpoints will be to assess long-term safety and tolerability of 20 mg BID of PF-02545920. Principal Investigator Lynn A. RaymondBody Locations and Systems Huntington's DiseaseArea VancouverAge 30-66
