Therapeutic Targets for Disorders of Human Neuromuscular Junction: A Scoping Review

The neuromuscular junction (NMJ) is a specialized connection between motor neurons and skeletal muscle fibers. NMJ dysfunction is a pathological hallmark of severe neuro-muscular and motor neuron diseases, including myasthenia gravis (MG), Lambert–Eaton myasthenic syndrome, congenital myasthenic syndrome, spinal muscular atrophy (SMA), and amyotrophic lateral sclerosis (ALS). Conventional pharmacotherapies, including ace-tylcholinesterase inhibitors and broad-spectrum immunosuppressants, provide symptomatic relief or nonspecific immune suppression that is often associated with significant side effects and limited efficacy. Overall, the treatment of NMJ disorders is shifting from nonspecific immunosuppression and symptom management toward precise, mechanism-based interventions. We follow PRISMA-ScR guidelines and involve database searches on Medline (via PubMed), Cochrane Central Register of Controlled Trials (CENTRAL), and Web of Science Core Collection between database inception and August 17, 2026. The aims of this project are collecting and synthesizing the available information and literature on current and emerging therapeutic targets for human NMJ disorders, and to describe the existing therapeutic landscape in a mechanism-based manner. The results will further explain the mechanisms of action of emerging therapies, and highlight remaining knowledge gaps and unmet therapeutic needs.