Dynamics of Onset and Progression in Amyotrophic Lateral Sclerosis DOI Creative Commons
Michael Swash, Mamede de Carvalho

Brain Sciences, Год журнала: 2025, Номер 15(6), С. 601 - 601

Опубликована: Июнь 3, 2025

This review focuses on the complexities of amyotrophic lateral sclerosis (ALS) onset, highlighting insidious nature disease and challenges in defining its precise origin early pathogenic mechanisms. The clinical presentation ALS is characterised by progressive muscle weakness wasting, often with widespread fasciculations, reflecting lower motor neuron hyperexcitability. disease’s pathogenesis involves a prolonged preclinical phase neuronal proteinopathy, particularly TDP-43 accumulation, which eventually leads to death overt ALS. discusses difficulties detecting this transition implications for therapeutic intervention. It also addresses involvement both upper systems, as well importance following presymptomatic patients genetic mutations. significance understanding distinct processes deposition subsequent degeneration developing effective treatments emphasised.

Язык: Английский

Neural Excitatory/Inhibitory Imbalance in Motor Aging: From Genetic Mechanisms to Therapeutic Challenges DOI Creative Commons
Xuhui Chen, Ya Wang, Yongning Zhang

и другие.

Biology, Год журнала: 2025, Номер 14(3), С. 272 - 272

Опубликована: Март 7, 2025

Neural excitatory/inhibitory (E/I) imbalance plays a pivotal role in the aging process. However, despite its significant impact, of E/I motor dysfunction and neurodegenerative diseases has not received sufficient attention. This review explores mechanisms underlying through lens balance, emphasizing genetic molecular factors that contribute to this (such as SCN2A, CACNA1C, GABRB3, GRIN2A, SYT, BDNF…). Key regulatory genes, including REST, vps-34, STXBP1, are examined for their roles modulating synaptic activity neuronal function during aging. With insights drawn from ALS, we discuss how disruptions balance pathophysiology age-related dysfunction. The genes discussed above exhibit certain association with neuron (like ALS), relationship had been previously recognized. Innovative therapies, such gene editing technology optogenetic manipulation, emerging promising tools restoring offering hope ameliorating deficits potential these technologies intervene aging-related diseases, challenges direct application human conditions.

Язык: Английский

Процитировано

0

Pathophysiology, Clinical Heterogeneity, and Therapeutic Advances in Amyotrophic Lateral Sclerosis: A Comprehensive Review of Molecular Mechanisms, Diagnostic Challenges, and Multidisciplinary Management Strategies DOI Creative Commons
Manuel González‐Sánchez, Marı́a Jesús Ramı́rez-Expósito, José Manuel Martı́nez-Martos

и другие.

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

Процитировано

0

Genetic architecture of amyotrophic lateral sclerosis: a comprehensive review DOI
Lamei Yuan,

Yuewen Yang,

Yi Guo

и другие.

Journal of genetics and genomics/Journal of Genetics and Genomics, Год журнала: 2025, Номер unknown

Опубликована: Май 1, 2025

Язык: Английский

Процитировано

0

Dynamics of Onset and Progression in Amyotrophic Lateral Sclerosis DOI Creative Commons
Michael Swash, Mamede de Carvalho

Brain Sciences, Год журнала: 2025, Номер 15(6), С. 601 - 601

Опубликована: Июнь 3, 2025

This review focuses on the complexities of amyotrophic lateral sclerosis (ALS) onset, highlighting insidious nature disease and challenges in defining its precise origin early pathogenic mechanisms. The clinical presentation ALS is characterised by progressive muscle weakness wasting, often with widespread fasciculations, reflecting lower motor neuron hyperexcitability. disease’s pathogenesis involves a prolonged preclinical phase neuronal proteinopathy, particularly TDP-43 accumulation, which eventually leads to death overt ALS. discusses difficulties detecting this transition implications for therapeutic intervention. It also addresses involvement both upper systems, as well importance following presymptomatic patients genetic mutations. significance understanding distinct processes deposition subsequent degeneration developing effective treatments emphasised.

Язык: Английский

Процитировано

0