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Lancet (2015) 386(9996):896912

Patient stories underscore the transformative role of these medications in igniting the potential for positive change, emphasizing the need for ongoing support and education to navigate the challenges of treatment

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10.3389/fonc.2020.00317 102 LingS.ZaccardiF.IssaE.DaviesM

Many other medications also use these pathways

The question sits within a broader debate over how much latitude Americans should have to access therapies that have not completed the controlled human trials ordinarily required before a drug reaches the market