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- FAIR Act could boost U.S. rare‑disease trial access and restore biotech leadershipIn a recent BioSpace podcast, host Jennifer C. Smith‑Parker talked with Max Bronstein of Aviva Strategies and Teresa Barnes of PF Warriors about a growing problem: U.S. patients are increasingly un…
- UniQure’s Huntington’s gene therapy shows reduced benefit in fourth year, but patient remains optimisticUniQure reported new data on its experimental Huntington’s disease gene therapy, AMT-130, in a four‑year follow‑up of 12 patients. The update showed the treatment slowed disease progression by 44%,…