Global research trends in biomarkers, therapeutic targets, and drugs for amyotrophic lateral sclerosis: a bibliometric and visualization analysis

Frontiers in Pharmacology, Год журнала: 2025, Номер 16

Опубликована: Апрель 7, 2025

Background Amyotrophic Lateral Sclerosis (ALS) is a fatal neurodegenerative disease characterized by progressive degeneration of motor neurons, marked complex pathological mechanisms and lack effective treatments. Despite substantial global research efforts, no comprehensive bibliometric analysis has systematically mapped the evolution ALS biomarkers, therapeutic targets, pharmacological advancements. Methods This study, based on 4,250 publications retrieved from Web Science Core Collection (2005–2025), employs tools such as CiteSpace VOSviewer to conduct first multidimensional trends in drug research. Results The results revealed contributions 20,168 authors across 92 countries, with annual growing at an average rate 16.5%. United States dominated output, accounting for 34.07% (n=1,448, TLCS=7,100), while Kingdom achieved highest impact 68 citations per article. Leading institutions, including University Oxford Milan, consistently produced high-impact studies. Pioneering scholars Turner MR Kiernan MC made significant advancing targets discovery. interdisciplinary integration molecular biology genetics emerged core driver progress Neurofilament light chain (NfL), antisense oligonucleotide (ASO) drugs, transcranial magnetic stimulation (TMS), oxygen free radicals (oxidative stress), gene therapy have remained central focuses field. Looking ahead, stem cell therapy, blood-brain barrier (BBB) penetration technologies, skeletal muscle targeting are poised emerge prominent directions. Conclusion dominates productivity, whereas demonstrates superior citation influence. China’s publication volume, its limited underscores necessity enhanced methodological rigor strategic international collaboration. Current priorities encompass NfL, TMS, ASO therapies, emerging innovations BBB technologies showing promise. Future directions should prioritize biomarker standardization, optimization delivery systems, Clinical Translation.

Язык: Английский

Pathophysiology, Clinical Heterogeneity, and Therapeutic Advances in Amyotrophic Lateral Sclerosis: A Comprehensive Review of Molecular Mechanisms, Diagnostic Challenges, and Multidisciplinary Management Strategies

Life, Год журнала: 2025, Номер 15(4), С. 647 - 647

Опубликована: Апрель 14, 2025

Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disorder characterized by the degeneration of upper and lower motor neurons, leading to muscle atrophy, paralysis, respiratory failure. This comprehensive review synthesizes current knowledge on ALS pathophysiology, clinical heterogeneity, diagnostic frameworks, evolving therapeutic strategies. Mechanistically, arises from complex interactions between genetic mutations (e.g., in C9orf72, SOD1, TARDBP (TDP-43), FUS) dysregulated cellular pathways, including impaired RNA metabolism, protein misfolding, nucleocytoplasmic transport defects, prion-like propagation toxic aggregates. Phenotypic manifesting as bulbar-, spinal-, or respiratory-onset variants, complicates its early diagnosis, which thus necessitates rigorous application revised El Escorial criteria emerging biomarkers such neurofilament light chain. Clinically, intersects with frontotemporal dementia (FTD) up 50% cases, driven shared TDP-43 pathology C9orf72 hexanucleotide expansions. Epidemiological studies have revealed lifetime risk 1:350, male predominance (1.5:1) peak onset 50 70 years. Disease progression varies widely, median survival 2–4 years post-diagnosis, underscoring urgency for intervention. Approved therapies, riluzole (glutamate modulation), edaravone (antioxidant), tofersen (antisense oligonucleotide), offer modest benefits, while dextromethorphan/quinidine alleviates pseudobulbar affect. Non-pharmacological treatment advances, non-invasive ventilation (NIV), prolong 13 months improve quality life, particularly bulb-involved patients. Multidisciplinary care—integrating physical therapy, support, nutritional management, cognitive assessments—is critical addressing non-motor symptoms dysphagia, spasticity, sleep disturbances). Emerging therapies show promise preclinical models. However, challenges persist translating insights into universally effective treatments. Ethical considerations, euthanasia end-of-life decision-making, further highlight need patient-centered communication palliative

Язык: Английский

Unlocking the future: Precision oligonucleotide therapy for targeted treatment of neurodegenerative disorders

International Journal of Biological Macromolecules, Год журнала: 2025, Номер 310, С. 143515 - 143515

Опубликована: Апрель 25, 2025

Язык: Английский