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- Ionis secures FDA approval for Zanvastro, first disease-modifying therapy for rare Alexander diseaseIonis Pharmaceuticals announced that the U.S. Food and Drug Administration has approved Zanvastro, marking the first disease-modifying treatment for Alexander disease, a rare and often fatal neurol…
- Ultragenyx to consider major cost cuts after Angelman drug fails trialUltragenyx announced that its experimental therapy GTX-102 (apazunersen) for Angelman syndrome failed to meet primary and secondary endpoints in a Phase 3 study. The company said there were no dif…