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- 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…
- Experts say Angelman syndrome drug failure leaves hope for similar antisense therapiesUltragenyx announced that its late-stage trial of an experimental Angelman syndrome drug did not meet its primary endpoints, marking a setback for the rare-disease community. Researchers such as M…