Ionis announces that FDA accepts New Drug Application and grants Priority Review of tofersen for a rare, genetic form of ALS

SOD1-ALS is a rare genetic form of ALS that affects approximately 330 people in the U.S. , it is progressive, leads to the loss of everyday functions and is uniformly fatal If approved, tofersen would be the first treatment to target a genetic cause of ALS 12-month data included in the filing show