Amyotrophic lateral sclerosis & frontotemporal degeneration最新文献

{"title":"Feature selection using metaheuristics to predict annual amyotrophic lateral sclerosis progression.","authors":"Thibault Anani, Jean-François Pradat-Peyre, François Delbot, Claude Desnuelle, Anne Sophie Rolland, David Devos, Pierre-François Pradat","doi":"10.1080/21678421.2025.2522399","DOIUrl":"https://doi.org/10.1080/21678421.2025.2522399","url":null,"abstract":"<p><strong>Objective: </strong>Amyotrophic lateral sclerosis (ALS), a progressive neurodegenerative disease with no curative treatment and affecting motor neurons, leads to motor weakness, atrophy, spasticity and difficulties with speech, swallowing, and breathing. Accurately predicting disease progression and survival is crucial for optimizing patient care, intervention planning, and informed decision-making.</p><p><strong>Methods: </strong>Data were gathered from the PRO-ACT database (4659 patients), clinical trial data from ExonHit Therapeutics (384 patients) and the PULSE multicenter cohort aimed at identifying predictive factors of disease progression (198 patients). Machine learning (ML) techniques including logistic/linear regression (LR), K-nearest neighbors, decision tree, random forest, and light gradient boosting machine (LGBM) were applied to forecast ALS progression using ALS Functional Rating Scale (ALSFRS) scores and patient survival over one year. Models were validated using 10-fold cross-validation, while Kaplan-Meier estimates were employed to cluster patients according to their profiles. To enhance the predictive accuracy of our models, we performed feature selection using ANOVA and differential evolution (DE).</p><p><strong>Results: </strong>LR with DE achieved a balanced accuracy of 76.05% on validation (ranging from 68.6% to 79.8% per fold) and 76.33% on test data, with an AUC of 0.84. With Kaplan-Meier's estimates, we identified five distinct patient clusters (<i>C</i>-index = 0.8; log-rank test <i>p</i> value ≤0.0001). Additionally, LGBM predictions for ALSFRS progression at 3 months yielded an RMSE of 3.14 and an adjusted <i>R</i>² of 0.764.</p><p><strong>Conclusion: </strong>This study showcases the potential of ML models to provide significant predictive insights in ALS, enhancing the understanding of disease dynamics and supporting patient care.</p>","PeriodicalId":72184,"journal":{"name":"Amyotrophic lateral sclerosis & frontotemporal degeneration","volume":" ","pages":"1-16"},"PeriodicalIF":0.0,"publicationDate":"2025-07-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144577104","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Lower-limb biomechanics in motor neuron disease: a joint-level perspective of gait disruption.","authors":"Cory J Holdom, James L Williamson, Georgia O'Reilly, Robert D Henderson, Sally Neville, Shyuan T Ngo, Taylor J M Dick, Frederik J Steyn","doi":"10.1080/21678421.2025.2523945","DOIUrl":"https://doi.org/10.1080/21678421.2025.2523945","url":null,"abstract":"<p><p><i>Background:</i> Motor neuron disease (MND) profoundly impacts mobility, yet gait-specific dysfunctions due to MND remain poorly understood. <i>Methods:</i> We characterized lower-limb gait alterations in people living with MND (plwMND) using advanced biomechanical tools. Nine plwMND and nine non-neurodegenerative disease controls walked on an instrumented treadmill at self-selected speeds. Ground reaction forces and joint motions were measured to model lower-limb kinetics, kinematics, and energetics. <i>Results:</i> PlwMND had reduced forward propulsive (<i>p</i> < 0.001) and braking (<i>p</i> = 0.002) ground reaction forces. Ankle range of motion was 10.0 ± 3.1° lower (<i>p</i> = 0.035) with peak positive ankle moment and power 33% and 72% lower, respectively (both: <i>p</i> < 0.001), in plwMND compared to controls. <i>Conclusions:</i> These lower-limb impairments highlight the ankle as an early and critical locus of dysfunction, with distal weakness driving compensatory proximal strategies, increasing walking inefficiency and fatigue. Integrating biomechanical and clinical data offers new insights into gait disruption in MND, supporting the development of targeted, personalized interventions to maintain independent mobility.</p>","PeriodicalId":72184,"journal":{"name":"Amyotrophic lateral sclerosis & frontotemporal degeneration","volume":" ","pages":"1-11"},"PeriodicalIF":0.0,"publicationDate":"2025-07-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144555980","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Prevalence and predictors of prolonged grief disorder, anxiety and depression in bereaved ALS family caregivers: a national survey of distress and support needs after bereavement.","authors":"Lone F Knudsen, Sabina Nikolajevic-Pujic","doi":"10.1080/21678421.2025.2523948","DOIUrl":"https://doi.org/10.1080/21678421.2025.2523948","url":null,"abstract":"<p><strong>Objective: </strong>The distress of amyotrophic lateral sclerosis (ALS) family caregivers may not end after bereavement. Yet, the prevalence of psychological distress and support needs after bereavement are unclear. This study examined the prevalence and predictors of prolonged grief disorder (PGD), anxiety and depression, and level of needed and received support after bereavement.</p><p><strong>Methods: </strong>We conducted a cross-sectional online survey of a national cohort of bereaved ALS family caregivers recruited through the National Rehabilitation Center for Neuromuscular Diseases in Denmark. Questions included disease-related factors, care involvement, burden (Zarit Burden Inventory), loneliness (Three-Item Loneliness Scale), coping style (CSQ-37), PGD (PG-13), anxiety/depression (HADS), time since bereavement, needed and received support during and after ALS.</p><p><strong>Results: </strong>A total of 162 caregivers (46.4%) with a median of 24 months since bereavement responded. PGD prevalence was 5.6%, anxiety 22.2%, depression 16.0%. PGD was predicted by more caregiving hours. Anxiety and depression by high emotional coping and not receiving the needed information post bereavement and, anxiety, also by a more recent bereavement. Half of the participants had needed information about ALS after bereavement with 17.4% receiving it to a small degree and 32.3% not at all. Nearly 80% had needed emotional support with 31.0% receiving it to a small degree/not at all.</p><p><strong>Conclusions: </strong>Caregivers may be distressed for a long time. Healthcare professionals should offer information about ALS to bereaved caregivers and screen caregivers for PGD, anxiety, depression, and coping style to offer targeted interventions post bereavement. Future longitudinal studies should investigate predictors for post-loss psychological distress including pre-loss anxiety/depression and formal care.</p>","PeriodicalId":72184,"journal":{"name":"Amyotrophic lateral sclerosis & frontotemporal degeneration","volume":" ","pages":"1-12"},"PeriodicalIF":0.0,"publicationDate":"2025-06-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144531357","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Quantitative susceptibility mapping for investigating brain iron deposits in amyotrophic lateral sclerosis: correlations with clinical phenotype and disease progression.","authors":"Maria Agnese Pirozzi, Fabrizio Canale, Federica Di Nardo, Minoo Sharbafshaaer, Carla Passaniti, Mattia Siciliano, Mario Cirillo, Alessandro Tessitore, Gioacchino Tedeschi, Fabrizio Esposito, Francesca Trojsi","doi":"10.1080/21678421.2025.2522406","DOIUrl":"https://doi.org/10.1080/21678421.2025.2522406","url":null,"abstract":"<p><p><i>Objective</i>: Perturbation of iron homeostasis is a potential key mechanism involved in neurodegeneration across many neurological disorders, including amyotrophic lateral sclerosis (ALS). We hypothesized that changes in quantitative susceptibility mapping (QSM) could capture perturbations in brain iron concentration in subgroups of ALS patients stratified by clinical phenotype and disease progression. <i>Method</i>: We enrolled 38 ALS patients (23 males - mean age: 58.7 ± 9.8), screened by clinical (ALS functional rating scale-revised, ALSFRS-R) and neuropsychological scales. Patients were a posteriori classified as fast (<i>n</i> = 16) or slow (<i>n</i> = 22) progressors. Two subgroups were also considered: pyramidal (or upper motor neuron+, UMN+) patients (<i>n</i> = 18), and patients with other phenotypes (<i>n</i> = 20). <i>Results</i>: Comparing fast vs. slow progressors, significant differences in iron deposits were observed in the left (<i>p</i> = 0.028) and right amygdala (<i>p</i> = 0.022), and in susceptibility distribution on the right hippocampus (<i>p</i> = 0.0011). Comparing UMN+ vs. other phenotypes, significant susceptibility differences emerged in the left thalamus (<i>p</i> = 0.0014) and right amygdala (<i>p</i> = 0.001). QSM changes were associated with baseline ALSFRS-R (rho = 0.36, p = 0.026) in the left paracentral cortex, and iron concentration with UMN score (rho = 0.35, <i>p</i> = 0.034). Moreover, the Edinburgh Cognitive and Behavioral ALS Screen (ECAS) was associated with iron deposits in the left thalamus (rho=-0.46, <i>p</i> = 0.0041). <i>Conclusions</i>: We confirmed that QSM alterations in extra-motor areas and subcortical regions may be distinctive hallmarks of neurodegeneration in pure/dominant UMN phenotypes of ALS. Moreover, we showed that QSM could be a valuable tool to differentiate patients with different progression rates and phenotypes, suggesting that QSM may support a prognostically useful early stratification of ALS patients.</p>","PeriodicalId":72184,"journal":{"name":"Amyotrophic lateral sclerosis & frontotemporal degeneration","volume":" ","pages":"1-9"},"PeriodicalIF":0.0,"publicationDate":"2025-06-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144531358","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A novel heterozygous variant of FUS gene associated with juvenile ALS and premature tremor onset: a case report.","authors":"Verónica Rodríguez-García, José Antonio Venta-Sobero, María Del Carmen Chima-Galán","doi":"10.1080/21678421.2025.2522403","DOIUrl":"https://doi.org/10.1080/21678421.2025.2522403","url":null,"abstract":"<p><p>Juvenile amyotrophic lateral sclerosis (JALS) is neurodegenerative disease of the upper and lower motor neurons of rare incidence. Although fused in sarcoma (FUS) mutations in JALS patients have been associated with movement disorders, here we described the case of a young girl with very early onset of tremor, years before commencement of weakness; once symptoms of JALS were stablished, a typical rapid disease progression from spinal to bulbar symptoms were noticed. Genetic testing revealed a novel mutation in FUS gene causative of a protein dysfunction. This case emphasizes the fact that some mutations within the FUS in JALS patients may produce a symptom onset with tremor.</p>","PeriodicalId":72184,"journal":{"name":"Amyotrophic lateral sclerosis & frontotemporal degeneration","volume":" ","pages":"1-3"},"PeriodicalIF":0.0,"publicationDate":"2025-06-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144478031","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Age and life stage in the experience of amyotrophic lateral sclerosis: a scoping review.","authors":"Andrea S E Parks, Lesley Gotlib Conn, Krystle Amog, Nicolas S Bodmer, James W King, Alexandra M R McLaren, Morgann Reid, Teruko Kishibe, Agessandro Abrahao, Lorne Zinman, Joanna E M Sale","doi":"10.1080/21678421.2025.2515914","DOIUrl":"https://doi.org/10.1080/21678421.2025.2515914","url":null,"abstract":"<p><p><i>Objective</i>: Understanding the experiences of people living with amyotrophic lateral sclerosis (plwALS) is necessary to appreciate their unique needs. Age and stage in the life course influence how illness is experienced; however, the extent to which age-specific complexities of living with ALS have been examined remains unexplored. This review aims to map the available evidence exploring age, age-graded role, or life-course transition with regards to the experience of ALS and to identify age-specific gaps in the literature. <i>Methods</i>: A scoping review guided by Joanna Briggs Institute methodology was undertaken. Eligible articles included peer-reviewed primary research studies, published in English from 2010 onward, investigating illness experience of adults with ALS with consideration for how age, age-graded roles, or life-course transitions influenced experience. Database sources included: Ovid's Medline, Embase, and PsycINFO; EBSCO CINAHL; and ProQuest Sociological Abstracts. Findings related to ALS experience and dimensions of age were summarized descriptively and categorized using qualitative content analysis. <i>Results</i>: Six thousand one hundred and eighty individual records were identified and screened. Forty-five articles, reporting 42 studies, were included. Findings regarding thoughts, feelings, or emotions of plwALS were most common and varied depending on whether they were in reference to chronological age or age-graded role. Despite the importance of life-course transitions for illness experience, they were not routinely considered. <i>Conclusion</i>: Numerous aspects of the experience of plwALS have been reported in reference to age; however, the significance of age-graded roles and life-course transitions warrants further examination. Recognition of age-related complexities of living with ALS will facilitate more personalized ALS care.</p>","PeriodicalId":72184,"journal":{"name":"Amyotrophic lateral sclerosis & frontotemporal degeneration","volume":" ","pages":"1-27"},"PeriodicalIF":0.0,"publicationDate":"2025-06-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144287397","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"ALSUntangled #79: alpha-lipoic acid.","authors":"Alaina Giacobbe, James Hiana, Olivia Wang, Michael Benatar, Paul Wicks, Javier Mascias Cadavid, Sartaj Jhooty, Christopher McDermott, Gary Pattee, Tulio Bertorini, Terry Heiman-Patterson, Dylan Ratner, Paul Barkhaus, Gregory Carter, Carlayne Jackson, Keelie Denson, Andrew Brown, Carmel Armon, Yuyao Sun, Andre Nguyen, Richard Bedlack, Xiaoyan Li","doi":"10.1080/21678421.2025.2507166","DOIUrl":"https://doi.org/10.1080/21678421.2025.2507166","url":null,"abstract":"<p><p>Alpha-lipoic acid (ALA) is a naturally occurring fatty acid. It serves as an essential cofactor for enzymatic reactions in mitochondrial energy production, is a potent antioxidant and has anti-inflammatory effects, which are plausible mechanisms in slowing ALS progression. In ALS preclinical studies, ALA slowed motor function decline and improved survival. There were self-reported cases of improved muscle strength in ALS patients when ALA was taken with numerous additional supplements, making it difficult to discern its efficacy. One small, 6-month open-label study showed improved quality of life, fatigue, and mood after participants took it with B vitamins and amino acids for the first 3 months. So far, no clinical trials have been published in people living with amyotrophic lateral sclerosis (PALS). Given the insufficient clinical data, we cannot endorse ALA and will support more research on its efficacy in slowing ALS progression.</p>","PeriodicalId":72184,"journal":{"name":"Amyotrophic lateral sclerosis & frontotemporal degeneration","volume":" ","pages":"1-5"},"PeriodicalIF":0.0,"publicationDate":"2025-05-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144136246","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"ALSUntangled #77: Psilocybin.","authors":"Bhavya Bakshi, Sandeep Yerraguntla, Carmel Armon, Paul Barkhaus, Tulio Bertorini, Robert Bowser, Sarah Breevoort, Mark Bromberg, Andrew Brown, Gregory T Carter, Vincent Chang, Jesse Crayle, Timothy Fullam, Maxwell Greene, Terry Heiman-Patterson, Carlayne Jackson, Sartaj Jhooty, Elise Mallon, Javier Mascias Cadavid, Christopher J Mcdermott, Gary Pattee, Kaitlyn Pierce, Dylan Ratner, Yuyao Sun, Olivia Wang, Paul Wicks, Martina Wiedau, Richard Bedlack","doi":"10.1080/21678421.2024.2441274","DOIUrl":"10.1080/21678421.2024.2441274","url":null,"abstract":"<p><p>ALSUntangled reviews alternate and off-label treatments prompted by patient interest. Here, we review psilocybin, a chemical derived from mushrooms and belonging in the category of drugs known as psychedelics. Psilocybin has plausible mechanisms for slowing ALS progression because of its ability to cross the blood brain barrier and effect neurogenesis and inflammation. Currently, there are no pre-clinical ALS models, case reports, or trials for psilocybin and ALS in the context of disease modifying therapy. Depending on dosing, there can be a high risk of psychological side effects including hallucinations and physical harm. Based on the above information, we do not currently support the use of psilocybin as a means to slow ALS progression.</p>","PeriodicalId":72184,"journal":{"name":"Amyotrophic lateral sclerosis & frontotemporal degeneration","volume":" ","pages":"385-388"},"PeriodicalIF":0.0,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142873640","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Characterization, epidemiology, and factors associated with evolution and survival in patients with amyotrophic lateral sclerosis in southeastern Spain, 2008-2021: a population-based study.","authors":"Yaiza García-Ramírez, Juana-María Cayuela-Fuentes, María-Pilar Mira-Escolano, Luis-Alberto Maceda-Roldán, Eva Mikulasova, Cristina Oliva-López, Antonia Sánchez-Escámez, Pilar Ciller-Montoya, Joaquín A Palomar-Rodríguez","doi":"10.1080/21678421.2024.2439454","DOIUrl":"10.1080/21678421.2024.2439454","url":null,"abstract":"<p><strong>Objective: </strong>To describe the epidemiology, characteristics, and factors associated with the evolution and survival in patients with amyotrophic lateral sclerosis (ALS) in a region of southeastern Spain.</p><p><strong>Methods: </strong>An observational study was carried out in people with a diagnosis of ALS in the period 2008-2021 who were registered in the Information System of Rare Diseases of the Region of Murcia (SIER). We calculated crude and standardized incidence rate (SIR) using European Standard Population of 2013 and point prevalence. The Kaplan-Meier method and the log-rank test were used to estimate and compare survival curves.</p><p><strong>Results: </strong>We identified 374 cases. The mean age at diagnosis was 66.5 ± 11.7 and 50.3% persons were spinal onset. Mean time from the onset of symptoms to diagnosis was 0.9 ± 1.0 years. The global SIR was 1.95/100,000 person-years (95%CI: 1.77-2.12), which was higher in men (ratio 1.34), and the point prevalence in 2021 was 4.57 per 100,000 (95% CI: 4.46-4.68). There were 297 deaths with a mean age of 69.8 ± 10.8. The median survival from clinical onset was 2 years (95%CI: 1.0-3.0). Factors associated with lower survival were bulbar onset (<i>p</i> < 0.001), older age at the onset of symptoms (<i>p</i> < 0.001), and the absence of riluzole treatment (<i>p</i> = 0.003).</p><p><strong>Conclusions: </strong>This study is one of few to evaluate the epidemiological, characteristics, and prognostic factors of ALS in Spain, with findings similar to previous population studies. The use of population-based registries offers reliable information on the magnitude, or evolution in these patients.</p>","PeriodicalId":72184,"journal":{"name":"Amyotrophic lateral sclerosis & frontotemporal degeneration","volume":" ","pages":"268-280"},"PeriodicalIF":0.0,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142900618","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Maximizing the translational potential of neurophysiology in amyotrophic lateral sclerosis: a study on compound muscle action potentials.","authors":"Scott McKinnon, Zekai Qiang, Amy Keerie, Tyler Wells, Pamela J Shaw, James J P Alix, Richard J Mead","doi":"10.1080/21678421.2024.2448540","DOIUrl":"10.1080/21678421.2024.2448540","url":null,"abstract":"<p><p>Mouse models of amyotrophic lateral sclerosis (ALS) enable testing of novel therapeutic interventions. However, treatments that have extended survival in mice have often failed to translate into human benefit in clinical trials. Compound muscle action potentials (CMAPs) are a simple neurophysiological test that measures the summation of muscle fiber depolarization in response to maximal stimulation of the innervating nerve. CMAPs can be measured in both mice and humans and decline with motor axon loss in ALS, making them a potential translational read-out of disease progression. We assessed the translational potential of CMAPs and ascertained time points when human and mouse data aligned most closely. We extracted data from 18 human studies and compared with results generated from SOD1^G93A and control mice at different ages across different muscles. The relative CMAP amplitude difference between SOD1^G93A and control mice in tibialis anterior (TA) and gastrocnemius muscles at 70 days of age was most similar to the relative difference between baseline ALS patient CMAP measurements and healthy controls in the abductor pollicis brevis (APB) muscle. We also found that the relative decline in SOD1^G93A TA CMAP amplitude between 70 and 140 days was similar to that observed in 12 month human longitudinal studies in APB. Our findings suggest CMAP amplitudes can provide a \"translational window\", from which to make comparisons between the SOD1^G93A model and human ALS patients. CMAPs are easy to perform and can help determine the most clinically relevant starting/end points for preclinical studies and provide a basis for predicting potential clinical effect sizes.</p>","PeriodicalId":72184,"journal":{"name":"Amyotrophic lateral sclerosis & frontotemporal degeneration","volume":" ","pages":"322-330"},"PeriodicalIF":0.0,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143017300","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}