Regeneron’s ultra-rare-disease drug won U.S. regulatory approval after a three-decade effort.
Experts say Neurocrine Biosciences’ newly approved Prader-Willi syndrome drug may be linked to a series of patient deaths.
STAT’s Pharmalittle warns that excluding rare-disease drugs from Medicare’s GUARD pilot could erase most planned savings, highlights MFN pricing pressure, and notes that AstraZeneca–Bristol Myers Squibb merger talks appear off.
An opinion piece arguing that regulatory tools meant to speed access to breakthrough treatments may be misused and hinder approvals, impacting LGMD patients.
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