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.