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 ...

Barth syndrome is a rare genetic disorder that mostly affects males and can cause problems with the heart, muscles, and immune system. Common symptoms of Barth syndrome include weak muscles, tiredness ...

Barth syndrome is a rare genetic disorder characterized by muscle weakness and heart abnormalities. The Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) for elamipretide ...

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 ...

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 ...

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 ...

Another ultra-rare therapy will soon be subject to the gaze of an FDA advisory committee, with the fate of more than 200 boys diagnosed with Barth syndrome worldwide in the balance. On Thursday, the ...

The U.S. Food and Drug Administration has granted accelerated approval to Forzinity (elamipretide) injection as the first treatment for Barth syndrome. Barth syndrome is a rare, serious, and ...

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, ...