Throughout 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 [...]
In 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. [...]
Gene 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 [...]
Health care decisions are never easy to make, especially when the decisions are complex such as considering a new therapy or participating in a clinical trial. Many health care professionals recognize this and are encouraging patients to play a more active role in making treatment decisions based on balancing evidence based options [...]
Amicus Therapeutics Announces European Commission Approval of Galafold® (migalastat) for Adolescents with Fabry Disease
The study, “Frequency of Fabry disease in a juvenile idiopathic arthritis cohort,” was published in the journal Pediatric Rheumatology.
AVROBIO Provides Regulatory Update on Investigational AVR-RD-01 for Fabry Disease
The Nordic Rare Disease Summit, organized as a virtual meeting on 12th and 13th of April 2021, gathered a wide range of rare disease experts, decision- and policymakers as well as representatives from NGO’s, patient organizations, academia and industry from across the Nordic countries
AVROBIO Provides Regulatory Update on Investigational AVR-RD-01 for Fabry Disease
The MPS - Mucopolisacaridosis y Síndromes Relacionados is organising their virtual international Fabry Congress 2021 on June 17-18, 18:00h. (CET)! They have an exciting program lined up with worldwide Fabry Experts in English and Spanish!
