This study primarily evaluates the safety, tolerability, and efficacy of RAG - 17 in adult ALS patients with SOD1 - mutated genes in the real - world setting.
18 ≤ Age ≤ 75 years old, regardless of gender.
ALS patients with confirmed SOD1 gene mutations (known SOD1 mutation sites with reported relevant disease progression).
The diagnosis meets the criteria for definite or probable familial or sporadic ALS in the El Escorial diagnostic criteria for amyotrophic lateral sclerosis revised by the World Federation of Neurology.
The patient himself/herself or their legal representative clearly understands, voluntarily participates in this study, and signs the informed consent form.
Patients with SOD1 mutation sites occurring at nucleotides 44 - 66 (counting from the start of SOD1 protein translation) and patients with P.F21C mutation.
Patients diagnosed with other mental illnesses according to the DSM - V diagnostic criteria, or those with obvious suicidal intent.
Patients with severe hepatic insufficiency, severe renal insufficiency, or severe cardiac insufficiency. (Severe hepatic insufficiency refers to an ALT value ≥ 2.0 times the upper limit of normal or an AST value ≥ 2.0 times the upper limit of normal; severe renal insufficiency refers to a CRE ≥ 1.5 times the upper limit of normal or an eGFR < 40 mL/min/1.73m²; severe cardiac insufficiency refers to a NYHA score of 3 - 4.)
Patients with a history of alcohol or drug abuse.