On the other hand, studies using shRNA-mediated TAFAZZIN knockdown in myeloid cell lines demonstrated increased caspase-3 activation, release of cytochrome c from mitochondria, and accelerated ...

On Thursday, the Cardiovascular and Renal Drugs Advisory Committee voted 10-6 that elamipretide is effective for the ultra-rare cardioskeletal disease caused by TAFAZZIN gene mutations.

In the briefing document, FDA reviewers said the agency "does not believe that the available evidence establishes the effectiveness of elamipretide" for Barth syndrome. The company's argument is ...

Barth syndrome is a rare, one in a million X-linked genetic disease of the Tafazzin (TAZ) gene, which impacts cardiolipin, an essential lipid in the mitochondria needed for energy creation.

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, ...

In people with Barth syndrome, however, a crucial mitochondria-housed gene, called tafazzin (TAZ), is mutated. Without TAZ, CL remodeling is stopped in its tracks and harmful lipids accumulate.

In people with Barth syndrome, however, a crucial mitochondria-housed gene, called tafazzin (TAZ), is mutated. Without TAZ, CL remodeling is stopped in its tracks and harmful lipids accumulate.

In people with Barth syndrome, however, a crucial mitochondria-housed gene, called tafazzin (TAZ), is mutated. Without TAZ, CL remodeling is stopped in its tracks and harmful lipids accumulate.