Through sheer determination, Neena Nizar has become the first patient in a clinical trial testing a treatment for her disease — one so rare it affects just 30 people worldwide.
Category: Rare and Orphan Diseases
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F.D.A. Turmoil Keeps Spotlight on Its Commissioner
The agency’s high-level turnover and conflicting policy decisions on drug oversight have fueled concerns about the leadership of Dr. Marty Makary.
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Drugmakers Notch a $5 Billion Win in Republicans’ Policy Bill
More medicines will be spared from Medicare price negotiations, a change that is projected to wipe out billions in savings for the federal government.