Ultragenyx slams the brakes on rare disease study after all 5 patients are hit with a serious setback
Ultragenyx $RARE and its partners at GeneTx have been forced to halt early-stage study for a drug to treat rare cases of Angelman syndrome after all 5 of the patients being treated for the first time experienced a severe adverse event at the highest dose.
The patients in the study experienced “lower extremity weakness believed to be related to local inflammation due to GTX-102,” triggering a red light on dosing and enrollment.
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