Targeted Therapy for Amyotrophic Lateral Sclerosis
A special issue of Journal of Clinical Medicine (ISSN 2077-0383). This special issue belongs to the section "Clinical Neurology".
Deadline for manuscript submissions: 10 November 2026 | Viewed by 90
Special Issue Editor
Interests: motor neuron diseases; amyotrophic lateral sclerosis; frontotemporal dementia; TDP-43; genetics; transcranial magnetic stimulation; magnetic resonance imaging
Special Issue Information
Dear Colleagues,
Amyotrophic lateral sclerosis (ALS) is a rapidly progressive neurodegenerative disorder characterized by the degeneration of upper and lower motor neurons and considerable clinical and molecular heterogeneity. ALS is clinically characterized by motor neuron dysfunction, and some patients also exhibit cognitive or behavioral deficits.
Advances in genetics and molecular neuropathology have reframed ALS as a clinical spectrum disorder whose pathogenesis is closely associated with the synergistic effects of multiple pathological mechanisms. These mechanisms also serve as the core targets for the exploration of targeted therapies in clinical practice. In clinicopathological studies, protein misfolding and aggregation, RNA metabolism defects, impaired nucleocytoplasmic transport, mitochondrial dysfunction, disrupted proteostasis and autophagy, excitotoxicity, axonal transport defects, and neuroinflammation have all been confirmed to correlate closely with the progression of clinical symptoms, disease severity, and prognosis in patients. Elucidation of these clinicopathological mechanisms provides important support for the development of targeted therapeutic strategies for ALS and drives the shift of treatment paradigms from symptomatic supportive care to disease-modifying therapy.
Emerging approaches include gene therapies currently under clinical investigation, pharmacological interventions targeting key clinical symptoms, immune modulators being evaluated in patient cohorts, and trials addressing biomarkers of disease activity and progression. This Special Issue focuses on advances in translating research findings based on clinicopathological mechanisms into disease-modifying therapies, thereby improving the clinical management of ALS.
Dr. Annemarie Hübers
Guest Editor
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Keywords
- amyotrophic lateral sclerosis
- targeted therapy
- motor neuron disease
- neurodegeneration
- disease-modifying therapy
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