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 ...

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.

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 ...

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 ...

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 has cleared a drug for the ultra-rare condition Barth syndrome, reversing an earlier decision to reject the therapy after a lengthy delay. The agency on Friday granted ...

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 ...

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 ...

RALEIGH, N.C. (WNCN) – Parents of children with ultra-rare diseases are encouraging the Food and Drug Administration to act quickly to approve drugs that could treat their conditions and to take into ...