The Board is responsible for the highest level of decision-making and communicates the organization’s vision to all members and the community.
The Annual WORLDSymposium is often cited as the most important scientific meeting on lysosomal molecular biology, disorders and treatment.
The Canadian Organization for Rare Disorders (CORD) is hosting a Rare Disease Day 2017 conference taking place on March 30-31, 2017.
The fifth Fabry International Expert Meeting is taking place on May 19 & 20, 2017 at the Royal Olympic Hotel in Athens, Greece.
Rare Disease Day takes place on the last day of February each year. Stay tuned for what YOU can do as a Canadian Fabry patient on this day.
The Phase I Gene Therapy Study for Fabry Disease is looking to recruit interested patients. This is a first-in-human study for the treatment of Fabry disease.
The BALANCE study is a clinical research study that will compare the safety and efficacy of an investigational medication ( a potential new medication) with an approved medication, agalsidase beta, also known as Fabrazyme.
Replagal (agalsidase alfa) was approved by Health Canada in 2004 and indicated for long-term enzyme replacement therapy in patients with a confirmed diagnosis of Fabry disease.
Amicus Therapeutics is currently developing migalastat, an oral drug designed to bind to and stabilize the α‐Gal A that is made in the patient’s own cells, with the intention of enabling its trafficking to lysosomes.