Biogen has unveiled Tofersen, a groundbreaking gene-targeted drug for amyotrophic lateral sclerosis (ALS) patients, marking a major step forward in the battle against this devastating disease in the Chinese mainland.
ALS progressively damages motor neurons in the brain and spinal cord, leading to muscle weakness, atrophy, and, in many cases, fatal respiratory failure within three to five years. The first injection was administered at Peking University Third Hospital, heralding the clinical availability of the Chinese mainland's first therapy that targets ALS caused by superoxide dismutase 1 (SOD1) gene mutations.
SOD1, the first gene linked to ALS, is also the most common pathogenic gene among ALS patients in the Chinese mainland. Typically emerging around the age of 50, SOD1-linked ALS affects both upper and lower motor neurons, often starting in the limbs.
Tofersen, an innovative antisense oligonucleotide (ASO), works by reducing the production of the toxic SOD1 protein, thereby slowing down motor neuron damage and disease progression 🧬. This breakthrough treatment brings renewed hope to patients and their families while inspiring future research into gene-targeted therapies 🚀.
Reference(s):
Breakthrough gene-targeted drug for ALS patients launched in China
cgtn.com
