The accelerated approval of elamipretide by the U.S. FDA marks a historic milestone in the treatment of mitochondrial diseases. It is the first therapy targeting cardiolipin dysfunction and the first approved drug for patients with Barth syndrome — an ultra-rare, life-threatening disorder that primarily affects boys.
Don‘t have an account? Create new account
Enter the email address that you registered with. We will send you instructions on how to set a new password.
