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- UniQure prepares for FDA advisory committee on Huntington’s gene therapy amid rare-disease regulatory jittersUniQure said it is ready to present its Huntington's disease gene therapy, AMT-130, to an FDA advisory committee, as discussed in its Q2 earnings call. The company expects the FDA may schedule the…
- Opinion: Limb girdle muscular dystrophy patients face a maddening reality“Too often, regulatory tools authorized to speed patient access to breakthrough treatments are being used in ways that even prevent approval,” writes Kat Bryant Knudson.