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 FDA rejected the company’s initial NDA in 2021, citing the lack of an adequate trial to demonstrate efficacy.
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Cushing's syndrome is fairly rare. It most often affects adults who are 20 to 50 years old. It is sometimes called hypercortisolism. You will probably need to see a specialist in hormone-related ...