New stroke treatment shows promise
There is currently only one drug approved for the condition, TPA, although it is only prescribed to a small fraction of patients and can also cause harmful side
Ultragenyx Pharmaceutical has received accelerated approval from the US Food and Drug Administration (FDA) for its gene therapy, Genglycos (pariglasgene brecaparvovec-opnr), to treat glycogen storage disease type Ia (GSDIa).