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- Industry urges FDA to broaden prior-knowledge guidance for gene and cell therapiesIndustry stakeholders have asked the U.S. Food and Drug Administration to expand the scope of its recent draft guidance on scientifically valid prior knowledge for gene therapies that involve genom…
- 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…