Core Viewpoint - The treatment for Amyotrophic Lateral Sclerosis (ALS) has entered a new era of precision intervention targeting pathogenic genes, with the successful administration of tofersen injection for SOD1 gene mutation patients in Guangzhou [1][3]. Group 1: Disease Overview - ALS, commonly known as "Lou Gehrig's disease," leads to progressive death of motor neurons in the brain and spinal cord, resulting in muscle weakness and atrophy, with a typical survival period of 3 to 5 years post-diagnosis [3]. - SOD1 is the most common pathogenic gene among ALS patients in China, with an average onset age of around 50 years, often starting from the lower limbs [3]. Group 2: Treatment Development - Tofersen injection, approved in China in September 2024, works by reducing the synthesis and accumulation of toxic SOD1 protein, thereby slowing down motor neuron damage [3]. - The rapid application of tofersen is attributed to the national rare disease prevention strategy and innovative drug policies, with the Guangzhou region's health insurance including it in their coverage [3]. Group 3: Industry Response - The emergence of breakthrough therapies has sparked hope among patients, as noted by leaders in the rare disease care community [4]. - The development and market introduction of tofersen injection have been positively acknowledged by organizations promoting the translation of scientific research into patient care [4].