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Monday, Apr 25 2016

Full Issue

Patients, Advocates To Flood FDA Panel Hearing On Experimental Muscular Dystrophy Treatment

The panel will hear from scientists and patients alike on Monday as it decides whether to recommend that the FDA approve the drug, called eteplirsen. More than 800 patient advocates have registered to appear, making it among the best attended FDA advisory committee meetings in history.

One of the most closely watched hearings on a proposed drug in years convenes Monday morning in Hyattsville, Md., where a panel of medical experts will consider an application by Cambridge’s Sarepta Therapeutics Inc. for approval of a Duchenne muscular dystrophy treatment. (Weisman, 4/25)

Billy Ellsworth, a teenager with an inexorable and devastating degenerative muscle disease, will bring a football with him to a Maryland hotel conference center on Monday. For months, he has been brainstorming a way to prove to a panel of scientists and physicians that the experimental drug he has been taking for more than four years has kept him strong and well — and he’d like to punctuate his brief testimony in the clearest possible way: by throwing them the ball. (Johnson, 4/23)

In other Food and Drug Administration news —

The U.S. Food and Drug Administration on Friday proposed a ban on electrical stimulation devices (ESDs) that are used to curb individuals from engaging in self-injurious or aggressive behavior, saying they pose an "unreasonable and substantial" risk to public health. (4/23)

Seven years after U.S. regulators slapped their strictest warning on two popular smoking-cessation medicines citing risks of suicidal behavior, a large international study found no such risk. (Johnson, 4/22)

This is part of the Morning Briefing, a summary of health policy coverage from major news organizations. Sign up for an email subscription.
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