Breakthrough gene-targeted drug for ALS patients launched in China

Biogen launched Tofersen, a groundbreaking gene-targeted drug for amyotrophic lateral sclerosis (ALS), in China on Tuesday. The first injection was administered at Peking University Third Hospital, marking the country’s first approved treatment for ALS caused by SOD1 gene mutations (SOD1-ALS), a common form in Chinese patients. Tofersen works by reducing toxic SOD1 protein production, slowing disease progression. Approved in China in September 2024, its commercial launch represents a major step in precision medicine for ALS, driven by national rare disease policies. Experts and patient advocates hailed the move as a milestone offering new hope for those affected.

Credit : CGTN