Focus on Fabry Leaders around the world
Charlotte2021-09-30T11:46:51+02:00Registration now open We are very pleased to invite you to our first online FIN Expert Meeting on April 24th, 2021!
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 [...]
Understanding the importance of shared health care decisions
Charlotte2021-09-30T11:37:51+02:00Health 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
Charlotte2021-09-16T16:08:20+02:00Amicus 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
Charlotte2021-09-16T16:04:41+02:00The study, “Frequency of Fabry disease in a juvenile idiopathic arthritis cohort,” was published in the journal Pediatric Rheumatology.
AVROBIO AVR-RD-01 Gene Therapy Trial Now Open to Female Patients
Charlotte2021-09-16T15:38:48+02:00AVROBIO Provides Regulatory Update on Investigational AVR-RD-01 for Fabry Disease
Nordic Rare Disease Summit
Charlotte2021-09-16T16:16:50+02:00The 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
Charlotte2021-06-18T15:52:30+02:00AVROBIO Provides Regulatory Update on Investigational AVR-RD-01 for Fabry Disease
MPS Spain – Fabry Congress 2021 on June 17-18, 18:00h
Charlotte2021-09-16T16:16:10+02:00The 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!
Open letter to Fabry Centres
Charlotte2021-09-30T12:24:53+02:00To highlight the need for continuous care and monitoring for Fabry disease patients and remind everyone of action needed for newly diagnosed patients, those in the midst of the diagnostic process or those experiencing symptoms for the first time and needing assessment We want to emphasise the need for ongoing clear and consistent communication from the centres about patients’ care and having regular appointments (in-person or telehealth)