Amyotrophic Lateral sclerosis (ALS) is a chronic neurodegenerative condition affecting nearly 18 million individuals globally. The current treatment options are not curative and are largely focused on controlling the progression of the disease. Its management is further complicated by the numerous pathophysiological factors associated with the condition. The USFDA recently granted accelerated approval to an American pharmaceutical company for its novel injectable drug called tofersen.
This drug has been approved for patients with mutations in the superoxide dismutase 1 gene (SOD1). The approval was based on the results of a 28-week study containing 108 ALS patients aged between 23-78 years. Study Details Double-blind, randomized, placebo-controlled trial Patients were grouped in a 2:1 ratio into a treatment and placebo group.. Patients in the treatment group had a 55% decrease in plasma…
