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.
The African Summit on Rare Diseases
Charlotte2022-02-08T16:37:17+01:00The African Summit on Rare Diseases 2021
News from Sangamo Therapeutics
Charlotte2021-11-04T17:15:33+01:00Sangamo Therapeutics Announces Preliminary Phase 1/2 Data Showing Tolerability and Sustained Elevated α-Gal A Enzyme Activity in Patients With Fabry Disease
Webinar: Fabry & the Brain
Charlotte2022-02-08T16:37:59+01:00Fabry Findings Webinar: Fabry & the Brain now available on our YouTube Channel
Fabry Outcome Survey- Annual Report 2020
Charlotte2022-02-08T16:37:44+01:00Throughout 2020, the COVID-19 pandemic posed many challenges for both FOS and the wider Fabry disease community. The members of the FOS Steering Committee and I would like to thank all patients and caregivers involved in the registry for their extremely valuable contributions and for their continued commitment to [...]
A new tool to capture symptoms for Fabry Disease?
Charlotte2021-09-30T12:02:34+02:00In December 2020 and March 2021, people with Fabry Disease, representing five European countries shared their perspectives on standard questionnaires often used to capture the experiences of people with Fabry Disease and the ideal profile of new tools, designed to more accurately record the Fabry Disease symptoms and impacts. [...]
What is Gene Therapy?
Charlotte2021-09-30T12:03:55+02:00Gene therapy is a way of altering the genetic instructions inside the body’s cells to treat or stop disease. Often, gene therapy works by introducing a correct copy of a defective gene into the patient’s cells, without removing or modifying the defective gene. Have a look at the infographic [...]