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Who gets to decide who receives experimental medical treatments?

For many patients, pushing for access to unproven treatments is their best chance of survival. And that’s worth the risk. ....

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FDA advisers recommend accelerated approval of a Sarepta gene therapy

The vote followed a daylong hearing, convened virtually, marked by debate over the company’s clinical trial data and impassioned pleas from the parents of boys with Duchenne, a deadly muscle-wasting disease. ....

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