News from Idorsia


Idorsia 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.

News from Idorsia2021-12-13T18:15:52+01:00

News from Sangamo Therapeutics


Sangamo Therapeutics Announces Preliminary Phase 1/2 Data Showing Tolerability and Sustained Elevated α-Gal A Enzyme Activity in Patients With Fabry Disease

News from Sangamo Therapeutics2021-11-04T17:15:33+01:00


Rare disease community calls for additional doses of COVID-19 vaccine to protect the most vulnerable and extra measures to vaccinate more European citizens


Fabry Outcome Survey- Annual Report 2020


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 [...]

Fabry Outcome Survey- Annual Report 20202022-02-08T16:37:44+01:00

A new tool to capture symptoms for Fabry Disease?


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. [...]

A new tool to capture symptoms for Fabry Disease?2021-09-30T12:02:34+02:00

What is Gene Therapy?


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 [...]

What is Gene Therapy?2021-09-30T12:03:55+02:00

Understanding the importance of shared health care decisions


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 [...]

Understanding the importance of shared health care decisions2021-09-30T11:37:51+02:00
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