Author name: Leonardo Arias

What the Leucovorin Approval Signals—or Doesn’t—About the FDA’s Rare Disease Stance

Although FDA Commissioner Marty Makary promised “an exciting treatment” for autism, what the agency delivered was a label expansion for leucovorin to treat the ultrarare cerebral folate deficiency. The regulatory process, which relied on a literature review rather than new evidence, stands in contrast to recent rare disease rejections in which the FDA cited a

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Astrocitos modificados para destruir las placas beta-amiloides de la enfermedad de Alzheimer

La enfermedad de Alzheimer, la principal causa de demencia en las poblaciones envejecidas de todo el mundo, se caracteriza por una cascada patológica distintiva: las placas de beta-amiloide se acumulan en el cerebro, desencadenando cambios perjudiciales en las proteínas tau, lo que en …

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