Ultragenyx Trial Failure Revives Debate About How to Treat a Rare Neuro Disorder

Ultragenyx Pharmaceuticals’ apazunersen did not meet the goals of its Phase 3 test in Angelman syndrome, a rare genetic neurological disorder with no FDA-approved therapies. The disappointing result has some readthrough to Ionis Pharmaceuticals and Oak Hill Bio, each in clinical development with Angelman drugs designed to work in a similar way as Ultragenyx’s failed molecule. The post Ultragenyx Trial Failure Revives Debate About How to Treat a Rare Neuro Disorder appeared first on MedCity News.