A Multicenter Open-Label Treatment Protocol to Observe the Safety of Replagal (Agalsidase Alfa) Enzyme Replacement Therapy in Canadian Patients With…The purpose of this study is to observe the safety of agalsidase alfa in Canadian patients with Fabry disease. Recruiting Principal Investigator Sandra SirrsArea Vancouver
Canadian Fabry Disease Initiative (CFDI) Enzyme Replacement Therapy (ERT) StudyCFDI STUDY with ENZYME REPLACEMENT THERAPY (ERT)Recruiting Principal Investigator Sandra SirrsBody Locations and Systems BrainArea VancouverAge 5-85
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. Recruiting Principal Investigator Anna LehmanBody Locations and Systems Brain and NervesArea VancouverAge 16-65 years
A Trial to Evaluate the Safety and Efficacy of Elamipretide in Subjects With Primary Mitochondrial Myopathy Followed by an Open-Label…The purpose of this study is to evaluate the safety and efficacy of daily subcutaneous injections of elamipretide in subjects with primary mitochondrial myopathy. Recruiting Principal Investigator Anna LehmanBody Locations and Systems Muscles, Neuromuscular DisordersArea VancouverAge 16-80
A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry DiseaseThe purpose of this study is to determine the long-term safety and tolerability of oral lucerastat in adult subjects with Fabry disease. Recruiting Principal Investigator Sandra SirrsArea VancouverAge 18 and above
