Interview with Julia Alton from the Canadian Fabry Association When did you join your national patient association? I was 18 years old when I joined the Canadian Fabry Association (CFA) as a youth representative. What was the reason for joining? At the time I recognized the need for reaching the Fabry [...]
Phase I/II clinical study exploring the potential of ST-920 investigational gene therapy to treat Fabry disease: recruiting participants now Fabry disease is caused by changes in a gene called GLA leading to the shortage of a protein called alpha-galactosidase A (α-Gal A). Isaralgagene civaparvovec, or ST-920, is an investigational gene therapy [...]
Interview with Manjit Singh from the Lysosomal Storage Disorders Support Society (LSDSS) India When did you join your national patient association? I started the patient organisation in 2006 which finally was formed formally and registered in 2010 by 10 like minded parents who all are the founding members. What was the [...]
Interview with Jack Johnson from FSIG (Fabry Support & Information Group) USA When did you join your national patient association? Well, since I founded the Fabry Support & Information Group with help from my mother and wife, it was from day one in 1996. What was the reason for joining? Forming [...]
Interview with Alejandra Tornero from Alianza Lisosomal Argentina When did you join your national patient association? I am the founder of Alianza Lisosomal Argentina – ALA – and I lead the Alliance for Patients with Fabry, Gaucher and Pompe diseases. What was the reason for joining? The reason I joined is [...]
On Rare Disease Day, the community joined together across borders and across diseases to show that; Rare is many. Rare is strong. Rare is proud! Here are some of the amazing activities our members organised!
Interview with Yifan Xu from Fabry China When did you join your national patient association? I joined Fabry China in 2016. At that time the community was only founded since a few months. There were only 10 members. You could hardly say it was an organization, actually more like just a [...]