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 ...

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.

Also in this episode, several Upstate and WNC families share their experiences with rare diagnoses. The Jones family is hosting an event in March to raise awareness for Fabry disease. Watch Part 1 ...