渐冻症治疗又有新突破：靶向治疗应用临床

Core Insights - The first commercial use of tofersen injection for treating SOD1-ALS in China has been officially launched, marking a significant milestone in targeted therapy for amyotrophic lateral sclerosis (ALS) [1][2] Group 1: Drug Approval and Mechanism - Tofersen injection received FDA approval in April 2023 and NMPA approval in September 2024, indicating its regulatory pathway in major markets [2] - The drug functions as an antisense oligonucleotide (ASO) that specifically binds to the SOD1 mRNA, which is linked to the disease [2] Group 2: Disease Background and Importance of Early Diagnosis - ALS, particularly SOD1-ALS, is characterized by the progressive degeneration of motor neurons, leading to muscle weakness and atrophy, with a typical survival time of 3-5 years post-diagnosis [2] - Early detection and treatment are crucial for patients with a clear genetic background, such as SOD1 mutations, emphasizing the need for awareness of symptoms like progressive muscle weakness and atrophy [2]