Two preliminary studies were published simultaneously in the New England Journal of Medicine, both aimed at reducing superoxide dismutase 1 (SOD1) protein levels in people who had amyotrophic lateral sclerosis (ALS) due to SOD1 mutations. In the first study, a phase I/II trial of intrathecal tofersen, Timothy Miller, MD, PhD, of Washington University in St. […]
Category: ALS Center News
Tofersen demonstrates positive results in Phase I/II trial for amyotrophic lateral sclerosis (Links to an external site)
Biogen (MA, USA) has announced positive results from a Phase I/II study of tofersen – an antisense oligonucleotide – for the potential treatment of SOD1 amyotrophic lateral sclerosis (ALS). These results have been published in The New England Journal of Medicine.
Experimental drug shows promising results for rare form of ALS (Links to an external site)
Researchers led by a Washington University neurologist found promising results for an experimental drug to treat a rare, inherited form of amyotrophic lateral sclerosis (ALS), commonly known as Lou Gehrig’s disease.
Research in St. Louis leads to promise in the fight against ALS (Links to an external site)
ST. LOUIS – Research taking place at Washington University’s School of Medicine is giving hope to those affected by amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease.
Experimental drug shows early promise against inherited form of ALS, trial indicates (Links to an external site)
Phase 1/phase 2 trial shows evidence for safety, biological activity of tofersen; phase 3 trial underway