Ultragenyx Trial Failure Revives Debate About How to Treat a Rare Neuro Disorder
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Frank Vinluan (via MedCity News)
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.
