Gobowen

Researchers are evaluating the long-term safety, tolerability, and effectiveness of GIVINOSTAT in patients with Duchenne Muscular Dystrophy (DMD) who have previously participated in GIVINOSTAT studies. This open-label study is designed for male patients aged 6 years and older and continues from prior treatments to monitor ongoing effects and safety. Participants receive GIVINOSTAT as an oral suspension (10 mg/mL), taken twice daily with food. The dose starts at the level used in their previous study and is adjusted based on weight and platelet counts to ensure safety. Platelet counts are closely monitored weekly during the first month and every two weeks during the second month, with specific criteria for dose reduction or permanent discontinuation if safety concerns arise. Throughout the study, patients undergo regular assessments including blood tests for safety markers and adverse event monitoring for about one year. The study tracks any treatment-related side effects and ensures appropriate follow-up until any issues resolve or stabilize. After study completion, participants have an end-of-study visit for final evaluations.