Timothy Miller, MD, PhD, talks with an amyotrophic lateral sclerosis (ALS) patient at Washington University School of Medicine in St. Louis. Miller led an international phase 3 clinical trial for a rare, inherited form of ALS. The trial, sponsored by the pharmaceutical company Biogen, showed that an investigational drug, known as tofersen, reduced molecular signs of the fatal disease, but at six months did not improve motor control and muscle strength. However, Miller and colleagues found evidence that longer-term use of the drug may help stabilize muscle strength and control.