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 continued approval of Forzinity is contingent upon verification and description of clinical benefit in a confirmatory trial. The Food and Drug Administration (FDA) has granted accelerated approval ...

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 ...

Ahead of an FDA advisory committee meeting on Thursday, agency reviewers cast doubt on the efficacy of elamipretide for Barth syndrome, an ultra-rare disease that has no approved treatments. In its ...

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 ...

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 ...

FDA advisors indicated a possible path forward for elamipretide as a treatment for Barth syndrome despite the lack of definitive evidence. On Thursday, the Cardiovascular and Renal Drugs Advisory ...

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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 ...