Badanie długoterminowego bezpieczeństwa i tolerancji lucerastatu u dorosłych z chorobą Fabry'ego

Fabry disease is a rare genetic disorder that affects the body's ability to break down a certain type of fat, leading to a buildup in various organs. This study focuses on the long-term safety and tolerability of a medication called lucerastat, which is taken orally in the form of a hard capsule. The main goal is to see how well people with Fabry disease can tolerate this medication over an extended period.

The study is designed as an extension of a previous trial and involves participants who have already completed a 6-month treatment period. During this study, participants will continue to take lucerastat to monitor any side effects or adverse reactions that may occur over time. The study does not include a control group or placebo, meaning all participants will receive the medication.

Participants will be monitored regularly to ensure their safety and to gather information on how the medication affects them in the long run. The study aims to provide valuable insights into the potential benefits and risks of using lucerastat for treating Fabry disease, helping to improve future treatment options for those affected by this condition.