If approved, this would be the first marketing authorization for elamipretide, a first-in-class mitochondria-targeted therapeutic, and the first FDA-approved therapy for Barth syndrome.

"We continue to work closely with the Agency as it completes its review of the elamipretide NDA and are actively preparing to support broad access to this therapy for individuals living with Barth ...

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.

Three months ago, the FDA’s Cardiovascular and Renal Drugs Advisory Committee voted 10-6 in support of elamipretide becoming the first approved treatment for Barth syndrome, an X-linked genetic ...

Barth syndrome (BTHS) is a rare genetic disorder primarily affecting males, characterized by a range of symptoms including cardiomyopathy, skeletal myopathy, and neutropenia. The condition is ...

Lyric Mariah Heard spoke with "Good Morning America" about her life as a model born with a rare birth defect and using humor ...

The medical community has been using people's names to label diseases, syndromes, and medical procedures for many years.