The recently launched phase 2 HELIOS clinical trial from Amylyx Pharmaceuticals, aims to explore the potential of AMX0035 (sodium phenylbutyrate and taurursodiol) in treating Wolfram syndrome, a rare and complex genetic disorder. In a recent interview, Drs. Lahar Mehta and Fumihiko Urano discussed the AMX0035 Wolfram syndrome trial design, objectives and implications for future research…

Cambridge, Massachusetts–based Amylyx has dosed the first participant in its phase 2 HELIOS clinical trial of AMX0035 focusing on Wolfram syndrome. The condition is an ultra-rare genetic disorder involving the endocrine system. Symptoms of Wolfram syndrome can include diabetes insipidus, diabetes mellitus, optic atrophy and hearing loss. In September, Amylyx won FDA approval for AMX0035…