source
- Ionis secures FDA approval for zilganersen, its first solo neurology therapy for Alexander diseaseIonis Pharmaceuticals announced that the U.S. Food and Drug Administration has granted approval to its investigational RNA‑based drug zilganersen for the treatment of Alexander disease, a rare neur…
- Ultragenyx's Angelman syndrome drug fails Phase 3 trial, halting developmentUltragenyx reported that its experimental antisense oligonucleotide designed for Angelman syndrome did not meet primary endpoints in a Phase 3 study. The trial, which enrolled patients with the ra…