Fabry's disease is an inherited condition that develops due to a mutation in the GLA gene. For most patients with this disease, the mutation causes the body to produce insufficient amounts of ...

As Rare Disease Day is commemorated on 28 February, experts discuss the barriers to gene therapy access for some rare ...

In May 2023, Elfabrio was approved by the FDA and EMA for Fabry disease. This is a $2 billion market dominated by three products [1] and one where legacy offerings present several shortcomings.