Drug development programs face unique challenges in demonstrating the safety and effectiveness of drugs for treating rare diseases. The FDA’s Accelerating Rare disease Cures (ARC) Program started the Learning and Education to...more
On April 25th, 2023, the U.S. Food and Drug Administration (FDA) approved QalsodyTM (tofersen) for the treatment of amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 (SOD1) gene...more