Freeline will be progressing to the second dose cohort in the MARVEL-1 study immediately with first patient dosing expected in mid-2022
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So far Charlotte has created 39 blog entries.
April 1st marks the start of Fabry Awareness Month. This year we focus our efforts on helping parents recognise Fabry symptoms in their children, with our campaign 'Cool Down Fabry'
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!