The U.S. Food and Drug Administration has approved Sephience (sepiapterin), an oral therapy by PTC Therapeutics, for the treatment of phenylketonuria (PKU), a rare inherited metabolic disorder that ...

The U.S. Food and Drug Administration has approved PTC Therapeutics' oral drug to treat a rare inherited metabolic disorder that prevents the body from properly breaking down certain amino acids, the ...

The US Food and Drug Administration (FDA) has approved PTC Therapeutics’ Sephience (sepiapterin) to treat the rare metabolic ...

The FDA approved sepiapterin (Sephience) for the treatment of children and adults with phenylketonuria (PKU), PTC Therapeutics announced. The approval includes broad labeling for the treatment of ...

By Dean Seal Shares of PTC Therapeutics climbed after the company said federal regulators approved its treatment for elevated levels of a certain amino acid in the blood. The stock was up 11%, at ...

The U.S. Food and Drug Administration has turned away PTC Therapeutics' proposed vatiquinone treatment for the rare, inherited neurodegenerative disorder Friedreich's ataxia over concerns about the ...

PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the U.S. Food and Drug Administration (FDA) has issued a ...