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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.
- Prolonged hospital stays leave children isolated, urging changeAn author visiting a pediatric ward describes a young, nonverbal child with severe developmental delays who has been waiting months for a life-saving liver transplant. The child lies in a stark, ma…