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. [...]
Fabry Findings Webinar - October 27th, 2021 - 5.30pm - 6.30pm CEST We are happy to invite you to the first Fabry Findings Webinar with Dr Simon Korver who will present the findings from our first issue.!
Registration now open We are very pleased to invite you to our first online FIN Expert Meeting on April 24th, 2021!
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
