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- FDA launches Rare Disease Framework to embed patient input early in drug developmentFDA's Center for Biologics Research and Evaluation (CBER) director Karim Mikhail announced a new Rare Disease Framework Initiative aimed at moving patient perspectives from an optional, late‑stage…
- New evidence links AAV gene therapies to tumor risk, raising safety concernsRecent cases reported this year provide strong evidence that adeno-associated viruses (AAVs), the vectors used to deliver therapeutic genes, can trigger tumor formation. The findings settle a two‑d…