Rare Neuro‑Inflammatory and Neuromuscular Disorders: Pathways to Precision Therapies

Rare neuro-inflammatory and neuromuscular disorders comprise a diverse group of conditions affecting the central and peripheral nervous systems and represent a major unmet need in neurology. Although clinically and biologically heterogeneous, emerging research is revealing meaningful mechanistic overlap across subsets of these disorders, including immune dysregulation, antibody- and complement-mediated injury, impaired neuromuscular transmission in selected conditions, and interactions between genetic susceptibility and environmental factors. Disorders within this scope include neuromyelitis optica spectrum disorder, myasthenia gravis, multifocal motor neuropathy, autoimmune encephalitides, hereditary neuropathies, rare ataxias, and rare motor neuron diseases. At the same time, these conditions retain important disease-specific features, underscoring the need for both cross-disease perspectives and disorder-specific investigation.This is a particularly timely moment for the field. Advances in molecular immunology, genomics, biomarker discovery, and targeted therapeutics are beginning to reshape how rare neurological disorders are classified, modeled, and treated. Complement inhibitors, FcRn antagonists, antisense oligonucleotides, and gene-based therapies have shown promise in selected disorders, illustrating the potential of mechanism-based and precision approaches across rare neurology. In parallel, patient-derived iPSC models, organoids, and other advanced experimental systems are creating new opportunities to interrogate disease biology, define heterogeneity, and improve prediction of therapeutic response.This Research Topic aims to provide a multidisciplinary forum for basic scientists, translational investigators, and clinicians working across rare neurology, neuroimmunology, and neuromuscular medicine. By bringing together mechanistic, experimental, and clinical studies across traditionally separate disease areas, it seeks to foster exchange of ideas, strengthen conceptual links across the field, and help bridge the gap between biological discovery and clinical impact. We welcome submissions that advance understanding of disease mechanisms and support the development of more precise diagnostic, prognostic, and therapeutic strategies for rare neuro-inflammatory and neuromuscular disorders.Topics of interest include immune dysregulation and neuroinflammatory signaling, complement and antibody-mediated mechanisms, genetic and molecular determinants of disease, biomarker discovery and patient stratification, translational models and experimental platforms, clinical phenotyping and natural history studies, precision therapeutics, and challenges in orphan drug development and access to emerging therapies.Topic Editor Dr. Thomas Theis is employed by Alexion, Rare Disease Unit of AstraZeneca. All other Topic Editors declare no competing interests with regards to the Research Topic subject. Topic editors: Thomas Theis, Suneel Kumar Source: https://www.frontiersin.org/research-topics/82303/rare-neuro-inflammatory-and-neuromuscular-disorders-pathways-to-precision-therapies