Core Insights - The article discusses the first administration of tofersen injection for ALS patients in Shandong, marking a significant milestone in the treatment of the disease in China [1][3]. Group 1: Drug Overview - Tofersen injection is the first approved drug targeting ALS patients with SOD1 gene mutations, representing a shift from symptomatic treatment to causal intervention [3][4]. - The drug is an antisense oligonucleotide (ASO) that reduces the synthesis of toxic SOD1 protein, thereby alleviating damage to motor neurons and slowing disease progression [4]. Group 2: Patient Demographics and Treatment - ALS, commonly known as "Lou Gehrig's disease," leads to the gradual death of motor neurons, resulting in muscle weakness and atrophy, with a typical survival time of 3-5 years [3]. - There are over 40,000 ALS patients in China, with more than 1,200 carrying the SOD1 gene mutation, which is the most common pathogenic gene in the Chinese ALS population [3]. Group 3: Accessibility and Cost - The cost of tofersen injection is approximately $199,200 (around 1.4 million RMB) for the first year, requiring 14 injections [6]. - To alleviate the financial burden on patients, the drug manufacturer has provided the medication free of charge to a charitable foundation for ALS patients [6]. Group 4: Genetic Considerations - Tofersen is specifically designed for patients with the SOD1 gene mutation, which only accounts for 2% of the ALS patient population [6]. - Genetic screening is recommended for patients with a family history of ALS to identify potential candidates for this targeted therapy [6].