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 [...]
Rare Diseases International Annual Membership Meeting
Fabry community leadership in an evolving landscape
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 [...]
International Fabry Congress MPS Lysosomales Association Spain
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 [...]
Help us to train Face2Gene to diagnose Fabry patients earlier!
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!
