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 ...

The U.S. Food and Drug Administration has granted accelerated approval to Forzinity (elamipretide) injection as the first treatment for Barth syndrome. Researchers at The Hospital for Sick Children ...

In a Nature Metabolism paper published today, researchers from the University of Pittsburgh detail a potential new target and a small-molecule drug candidate for treating Barth syndrome, a rare, ...

No revised PDUFA target date has been communicated by the FDA. A decision on the New Drug Application for elamipretide for the treatment of Barth syndrome has been delayed again, according to Stealth ...

Boys suffering from Barth syndrome, a genetic determined disease with a very high mortality rate, finally have hope for a cure. What has contributed to this finding is research of Dr. habil Karolina ...

The U.S. Food and Drug Administration has granted accelerated approval to Forzinity (elamipretide) injection as the first treatment for Barth syndrome. Researchers at The Hospital for Sick Children ...

The Food and Drug Administration (FDA) has accepted for review the resubmitted New Drug Application (NDA) for elamipretide in the treatment of Barth syndrome, a rare mitochondrial disease.

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 ...

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 ...

Researchers at The Hospital for Sick Children (SickKids) have uncovered a promising new therapeutic target for Barth syndrome, a rare genetic condition with no current cure. Barth syndrome is an ...

ORLAND PARK, Ill. (WLS) -- A baby in the Chicago area is battling a rare disease and desperately needs a drug treatment for a better life. The drug has now been granted accelerated approval by the FDA ...

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