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- 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.
- Alnylam shares tumble nearly 30% after cutting revenue outlook for key amyloidosis drugsAlnylam Pharmaceuticals saw its market value drop about 30%, roughly $12 billion, after it lowered its revenue forecast for its leading amyloidosis treatments. The company had previously expected…