Help us to train Face2Gene to diagnose Fabry patients earlier!
Charlotte2022-03-31T13:31:22+02:00Help us to train Face2Gene to diagnose Fabry patients earlier!
Rare Disease Day 2022
Charlotte2022-03-16T15:45:13+01:00On 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!
News from Sangamo Therapeutics
Charlotte2022-02-08T16:29:44+01:00Sangamo Therapeutics Announces Updated Preliminary Phase 1/2 Data Showing Tolerability and Sustained Elevated α-Gal A Enzyme Activity in Patients With Fabry Disease
Webinar Fabry Highlights of the WorldSymposium 2023
Charlotte2023-04-07T16:01:37+02:00Join us for the Fabry Highlights from the Worldsymposium Webinar with Dr Hopkin April 5th, 2023 at 6.30pm-7.30pm CET
Fabry Expert Meeting 2022 – Virtual on May 7th
Charlotte2022-03-11T10:42:28+01:00Virtual Fabry Expert Meeting 2022 on May 7th
Addressing the Challenges of Persons Living with a Rare Disease
Charlotte2022-02-08T16:39:01+01:00Addressing the Challenges of Persons Living with a Rare Disease
Gene Therapy Approaches for Fabry Diseases
Charlotte2022-02-08T16:39:13+01:00We'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!
News from Freeline
Charlotte2021-12-13T18:27:08+01:00Freeline 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.
News from Idorsia
Charlotte2021-12-13T18:15:52+01:00Idorsia 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.