The US Food and Drug Administration (FDA) has delayed its decision on Stealth BioTherapeutics’ new drug application (NDA) for elamipretide, a treatment for the ultra-rare disease Barth syndrome.

The following day, National Donor Day, Devin underwent his second transplant. After some time to recover, he was on his way ...

The FDA is expected to decide on treatments for dry eye disease, neuroendocrine tumors, chronic spontaneous urticaria, Barth syndrome, and migraine.

Addington was diagnosed with Barth syndrome at two years old, a rare genetic disorder that primarily affects boys. Barth syndrome causes heart problems, muscle weakness, growth delays, and a ...