STAT+: Ionis wins FDA approval for first drug for Alexander disease, a rare and deadly neurological disorder

Ionis Pharmaceuticals on Thursday received Food and Drug Administration approval for the first disease modifying therapy for Alexander disease, a rare and deadly neurological condition.

The FDA approved the drug, Zanvastro, for use in children and adults after a pivotal trial showed that walking speed, a common measure of motor function in neurologic disease, stayed stable in treated patients while those in the control group saw a 33% decline. 

The study also contained hints that treating young children can improve motor function, not just stabilize it. Researchers found that the drug was generally safe, with serious adverse events more common in the control group than among those treated.

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