Barth Syndrome is a rare genetic disorder caused by an abnormality in the X chromosome. The condition is inherited, but is almost exclusively found in male children as the X chromosome is passed from ...

Follows Positive FDA Advisory Committee Meeting Vote Concluding that Elamipretide is Effective for the Treatment of Barth Syndrome NEEDHAM, Mass., Jan. 23, 2025 /PRNewswire/ -- Stealth BioTherapeutics ...

SAN FRANCISCO (KPIX) — With the FDA recently rejecting a drug application that aims to help those living with Barth Syndrome, one Bay Area family is frustrated by the delay of approved treatments in ...

Saturday marks National Rare Disease Day to bring awareness to some of the world's most unique conditions.Mobile users tap here for videoA disease is defined as rare when it affects less than 200,000 ...

NEW YORK (WABC) -- People with Barth Syndrome - an ultra-rare, life-threatening disease - and parents of children with it are pushing the Federal Drug Administration merely to review an application ...

BOSTON, April 29, 2025--(BUSINESS WIRE)--The Barth Syndrome Foundation (BSF), the only patient advocacy organization solely dedicated to saving lives through education, advances in treatments, and ...

Barth syndrome is a rare, X‐linked genetic disorder predominantly caused by mutations in the TAZ gene, which encodes the enzyme tafazzin. Tafazzin is responsible for the remodelling of cardiolipin, a ...

NEEDHAM, Mass., Jan. 23, 2025 /PRNewswire/ -- Stealth BioTherapeutics Inc. (the "Company" or "Stealth"), a clinical-stage biotechnology company focused on the discovery, development and ...