For anyone wondering why Americans disgusted with government would take a flyer on Donald Trump, consider Monday at the Food and Drug Administration: A panel of experts recommended denying young boys with a lethal form of muscular dystrophy access to an experimental drug with four years of promising clinical results.
A 13-member FDA advisory committee voted against approving eteplirsen, a treatment for Duchenne muscular dystrophy by Sarepta Therapeutics, concluding that there wasn’t sufficient evidence the drug was effective. Some 800 physicians, patients and parents beg to differ and showed up at the 11-hour hearing in Maryland. FDA can follow or ignore the committee and is set to decide by May 26.
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