Continued access to lucerastat for Fabry disease

This trial tests a new medicine called lucerastat for men with Fabry disease who have not yet received treatment. The goal is to see if it can slow down or improve symptoms and whether it is safe.

This study looks at how well available Fabry disease treatments (like migalastat or ERT) work over time in real life. It may help doctors understand who benefits most from these medicines.

This expanded access program provides migalastat (a pill) to people with Fabry disease who have a specific genetic mutation and cannot use standard enzyme replacement therapy. It's an option for those who need treatment and have no other alternatives.

This trial tests if enzyme replacement therapy works well and is safe for Fabry disease. It is for people who are already prescribed this treatment.

This trial tests if the oral drug migalastat is safe and works for children aged 2 to under 12 with a specific type of Fabry disease. It may be an option if your child has not been on enzyme therapy recently and has a compatible gene variant.

This Phase 1/2 study tests how safe and potentially effective 4D-310 is for adults with Fabry disease who have heart involvement. It may help people who can’t tolerate standard treatments like enzyme replacement therapy (ERT) or migalastat, or who still have disease progression.