In an open-label extension of a Phase III clinical trial sponsored by the pharmaceutical company Biogen, investigators have shown that the use of the drug tofersen for one year may help stabilize muscle strength and motor control in patients with an inherited form of amyotrophic lateral sclerosis (ALS), or Lou Gehrig’s disease. At only six months, however, tofersen did not improve clinical symptoms, even though molecular signs of the disease were improved.
