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- FDA approves Zanvastro, first therapy targeting protein buildup in Alexander diseaseThe U.S. Food and Drug Administration has approved Zanvastro (zilganersen) injection for the treatment of Alexander disease in both pediatric and adult patients. Zanvastro is the first FDA-approve…
- Ultragenyx’s Angelman therapy fails Phase 3, stock drops over 43% and company announces strategic reviewUltragenyx reported that its investigational Angelman syndrome drug apazunersen did not meet the primary cognitive endpoints in a Phase 3 trial. The lack of efficacy means the therapy will not prov…