Oral Ifetroban in Subjects With Duchenne Muscular Dystrophy
Status:
Recruiting
Trial end date:
2023-07-01
Target enrollment:
Participant gender:
Summary
Duchenne muscular dystrophy (DMD) is a devastating X-linked disease which leads to loss of
ambulation between ages 7 and 13, respiratory failure and cardiomyopathy (CM) at any age, and
inevitably premature death of affected young men in their late twenties. DMD is the most
common fatal genetic disorder diagnosed in childhood. It affects approximately 1 in every
3,500 live male births across all races and cultures, and results in 20,000 new cases each
year worldwide.Significant advances in respiratory care have unmasked CM as the leading cause
of death. As there are yet no specific cardiac treatments to extend life, the current study
aims to address this unmet medical need using a new therapeutic strategy for patients with
DMD.
Funding Source - FDA OOPD