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 ...