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!
Sangamo Therapeutics Announces Updated Preliminary Phase 1/2 Data Showing Tolerability and Sustained Elevated α-Gal A Enzyme Activity in Patients With Fabry Disease
Join us for the Fabry Highlights from the Worldsymposium Webinar with Dr Hopkin April 5th, 2023 at 6.30pm-7.30pm CET
Virtual Fabry Expert Meeting 2022 on May 7th
Addressing the Challenges of Persons Living with a Rare Disease
We're are happy to share this great resource with you. This can support you in exploring different gene therapies approaches to treating Fabry disease! Have a look at the infographic to find out more!
Freeline announced new data from its ongoing Phase 1/2 MARVEL-1 dose-finding clinical trial of FLT190 for the treatment of Fabry disease and provided updates on its pipeline programs.
Idorsia Ltd (SIX: IDIA) today announced that after the planned interim analysis of the open-label extension (OLE) of the Phase 3 MODIFY study with lucerastat for the treatment of adult patients with Fabry disease, the study will continue.
The African Summit on Rare Diseases 2021
