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. […]
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.
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.
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.
Phase 1/phase 2 trial shows evidence for safety, biological activity of tofersen; phase 3 trial underway