The top 10 orphan drugs in the late-stage pipeline
Orphan drugs have come into the spotlight, scaring up headlines involving big prices and small patient populations. Some companies have been accused of profiteering by manipulating the FDA’s rules on this — hello, Marathon — but orphan therapies remain a very closely watched arena with some major new prospects coming through the late-stage pipeline. And some of these drugs are offering patients real hope for the first time.
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