The FDA approved Sephience - PTC's oral therapy for a rare metabolic disorder called phenylketonuria - last month, ...

PTC Therapeutics has long been trying to workshop its vatiquinone into a marketable treatment for Friedreich's ataxia (FA) after a phase 3 fail in 2023. | Despite missing its primary endpoint in a ...

The International Progressive MS Alliance announces the launch of its MS Clinical and Imaging Data Resource (CIDR), which ...