Core Viewpoint - The innovative drug Tofersen injection for treating SOD1-ALS has officially launched in China, marking a significant advancement in the treatment of this rare and fatal neurological disease [1][2]. Group 1: Drug and Treatment Details - Tofersen injection is the first disease-modifying treatment for SOD1-ALS, providing a new hope for patients suffering from this condition [1][2]. - The drug is an antisense oligonucleotide (ASO) that reduces the synthesis of the toxic SOD1 protein, thereby alleviating damage to motor neurons and slowing disease progression [2][3]. - The drug was approved in September 2022 for adult patients with SOD1 gene mutations, addressing a significant unmet need in the treatment of ALS [2][3]. Group 2: Patient and Clinical Insights - The average onset age for SOD1-ALS patients is around 50 years, with a typical presentation of lower limb onset and simultaneous damage to upper and lower motor neurons [3]. - There is an urgent demand for targeted therapies among patients due to the fatal nature of the disease [3]. - The importance of genetic testing for all ALS patients, including those with SOD1 mutations, is emphasized for accurate diagnosis and treatment [2][3]. Group 3: Advocacy and Future Directions - Experts call for the standardization of genetic testing and treatment protocols to ensure timely access to this breakthrough therapy for eligible patients [3]. - There is a collective appeal from advocacy groups to raise awareness and improve access to innovative therapies for rare disease populations [3].

Core Viewpoint - The article discusses the promising results of a clinical trial that tested low-dose IL-2 as an add-on therapy to Riluzole for treating Amyotrophic Lateral Sclerosis (ALS), indicating that modifying the immune system may effectively slow disease progression and reduce mortality risk [2][4][9]. Group 1: Disease Overview - Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig's disease, is a rare neurodegenerative disorder characterized by the progressive loss of motor neurons, leading to severe disability and a median survival time of only 2-3 years from symptom onset [1]. - Current treatment options are limited, with Riluzole being the only FDA-approved drug that can extend survival by only a few months, highlighting the urgent need for more effective therapies [1]. Group 2: Clinical Trial Details - A phase 2b, double-blind, randomized, placebo-controlled trial was conducted to evaluate the efficacy and safety of low-dose IL-2 as an adjunct therapy to Riluzole in ALS patients [2][4][5]. - The trial included participants aged 18-76 with a symptom duration of ≤24 months and a forced vital capacity of ≥70%, who had not previously received Riluzole treatment [5]. Group 3: Trial Results - Out of 304 screened ALS patients, 220 met the randomization criteria after a 12-18 week Riluzole monotherapy period, with a 62% male and 38% female distribution [6]. - The primary endpoint analysis indicated a 19% reduction in mortality risk for the IL-2 LD group, although this was not statistically significant; however, a subsequent adjusted analysis showed a significant 68% reduction in mortality risk [6][7]. - The treatment was found to be safe, with significant increases in Treg cells and decreases in plasma CCL2 levels observed at all time points [6][8]. Group 4: Implications for Treatment - The findings suggest that low-dose IL-2 could be considered a safe and well-tolerated treatment option for ALS, enhancing the effects of Riluzole and potentially improving patient survival rates [2][9].