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.

Barth’s syndrome in an inherited disorder in an X-linked fashion. The cardiac component includes dilated cardiomyopathy which can at times cause severe systolic congestive heart failure.

The latest research report published by Vantage Market Research, titled "An Increase in Demand and Opportunities for the Global Barth Syndrome Treatment Market 2025-2035," provides a comprehensive ...

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 ...

Barth syndrome is caused by a mutation in the tafazzin gene, which results in reduced cardiolipin levels, a phospholipid that plays a major role in mitochondrial function. The disease is characterized ...

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

She is also a graduate of the Hearst Leadership Institute. Stacey currently serves as fundraising chairperson for the Barth Syndrome Foundation, a nonprofit advocacy organization that helps people ...

The FDA is expected to decide on treatments for dry eye disease, neuroendocrine tumors, chronic spontaneous urticaria, Barth syndrome, and migraine. Reproxalap, a first-in-class small-molecule ...