Muscle & Nerve最新文献

{"title":"Prevalence of Pathogenic Transthyretin Gene Variants in the Rocky Mountain Region.","authors":"Ellie Jacoby, Dianna Quan, Emily Todd, Jonathan Shortt, Harry Smith, Nicholas Rafaels, Kristy Crooks","doi":"10.1002/mus.28301","DOIUrl":"10.1002/mus.28301","url":null,"abstract":"<p><strong>Introduction/aims: </strong>Hereditary transthyretin amyloidosis (ATTRv) is a genetic condition caused by pathogenic variants in the transthyretin (TTR) gene resulting in multisystem amyloid deposition, especially in peripheral nerve and heart. Information on the prevalence of ATTRv in the United States is limited. The objective of this study was to understand the prevalence and genetic ancestry in the Val142Ile population in a large regional US population.</p><p><strong>Methods: </strong>Colorado Center for Personalized Medicine (CCPM) biobank collects specimens from consenting adults seen throughout the University of Colorado Health System, a large tertiary healthcare system within the Rocky Mountain Region (RMR). Single nucleotide polymorphism (SNP) array genotyping and whole exome sequencing (WES) were performed for genetic research. Prevalence of TTR variants was studied.</p><p><strong>Results: </strong>A total of 73,346 participants had genetic testing; 151 (0.21%) individuals had a pathogenic or likely pathogenic TTR variant. The most common variant was Val142Ile, making up 82.61% of TTR variants found. Over four percent of people with African non-Hispanic ancestry had a Val142Ile variant.</p><p><strong>Discussion: </strong>The most common TTR variant in our study was Val142Ile, the most common variant in the United States (US). Similar to other US-based studies, affected individuals were predominantly of African, non-Hispanic ancestry. The availability of treatments for symptomatic ATTRv patients raises opportunities and challenges for biobanks as the identification of at-risk individuals places pressure on highly specialized centers and providers to see patients for screening and follow-up.</p>","PeriodicalId":18968,"journal":{"name":"Muscle & Nerve","volume":" ","pages":"252-256"},"PeriodicalIF":2.8,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142801713","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Sarcolemmal Excitability Properties of the Trapezius.","authors":"Mitchell J Lycett, James Lee, Robert Boland-Freitas, Karl Ng","doi":"10.1002/mus.28361","DOIUrl":"https://doi.org/10.1002/mus.28361","url":null,"abstract":"<p><strong>Introduction/aims: </strong>The sarcolemma can be probed in vivo using muscle excitability recordings. These techniques have been applied to the tibialis anterior and brachioradialis muscles in healthy and diseased states. We aimed to study and validate the trapezius as a more proximal muscle to be used for myopathic conditions.</p><p><strong>Methods: </strong>Muscle velocity recovery cycles (MVRCs) and frequency ramp recordings were performed on the trapezius of 29 healthy controls. These recordings were compared to tibialis anterior recordings from 74 healthy controls.</p><p><strong>Results: </strong>Overall, the recorded MVRC and frequency ramp recordings were similar between the trapezius and tibialis anterior. The mean relative refractory period (MRRP) was shorter in the trapezius (3.29 vs. 3.71 ms), but this is explained by differences in muscle temperature.</p><p><strong>Discussion: </strong>The trapezius demonstrates similar MVRC and frequency ramp parameters to the more commonly studied tibialis anterior. The trapezius is technically easy to study using muscle excitability techniques and therefore presents itself as a promising muscle to study sarcolemmal properties in conditions that preferentially affect the proximal musculature.</p>","PeriodicalId":18968,"journal":{"name":"Muscle & Nerve","volume":" ","pages":""},"PeriodicalIF":2.8,"publicationDate":"2025-01-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143066581","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Orbicularis Oculi Stimulated Jitter Analysis in Children With Autoimmune Myasthenia Gravis.","authors":"Vishva Natarajan, Sumit Verma","doi":"10.1002/mus.28363","DOIUrl":"https://doi.org/10.1002/mus.28363","url":null,"abstract":"<p><strong>Introduction/aims: </strong>Stimulated jitter analysis (stim-JA) involves analyzing the variation in time intervals between stimulations and action potentials, expressed as the mean consecutive difference (MCD). The MCD upper limits are derived from adult populations and warrant a re-evaluation in children to accurately diagnose neuromuscular junction (NMJ) defects.</p><p><strong>Methods: </strong>A retrospective chart review was conducted which analyzed orbicularis oculi stim-JA studies performed on children between January 2014 and December 2021. The clinical profile, acetylcholine receptor (AChR), and muscle-specific kinase (MuSK) antibody status as well as stim-JA study results were retrieved. Bootstrapping was applied to the stim-JA studies to derive de novo MCD upper limits.</p><p><strong>Results: </strong>Twenty-seven stim-JA studies were performed on patients aged 3-19 years with either definite myasthenia gravis presentations and NMJ defects (n = 19, 17 AChR+, 2 MuSK+) or those with normal neurological examinations who were seronegative (n = 8). Four hundred ninety-nine apparent single fiber action potentials (ASFAPs) were analyzed with the individual and mean MCD significantly higher in children with autoimmune myasthenia (p < 0.05). Bootstrapping analysis revealed that MCD upper limits of 39 μs for individual MCD and 24 μs for mean MCD significantly improved specificity (p < 0.05) while maintaining sensitivity of the test in distinguishing definite MG NMJ defect from normal NMJ function.</p><p><strong>Discussion: </strong>Stim-JA using revised upper limits may help clinicians avoid the over-diagnosis of NMJ disorders in children. Stim-JA is a safe and well-tolerated method to screen for definite MG in children over 2 years.</p>","PeriodicalId":18968,"journal":{"name":"Muscle & Nerve","volume":" ","pages":""},"PeriodicalIF":2.8,"publicationDate":"2025-01-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143066556","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Clinicopathological Features of Mixed Connective Tissue Disease-Related Myositis: A Case Series.","authors":"Naohiro Sakamoto, Rumiko Izumi, Naoki Suzuki, Maki Tateyama, Masashi Aoki","doi":"10.1002/mus.28360","DOIUrl":"https://doi.org/10.1002/mus.28360","url":null,"abstract":"<p><strong>Introduction: </strong>Mixed connective tissue disease (MCTD) patients often have myositis, however, myopathological and clinical data for MCTD are limited. Recent reports have shown that the pathology of MCTD myositis resembles that of immune-mediated necrotizing myopathy (IMNM), whereas earlier reports described perifascicular atrophy or inflammatory infiltrates predominantly in the perivascular region in MCTD myositis. We aim to describe the clinical and myopathological features of MCTD myositis.</p><p><strong>Methods: </strong>We analyzed the clinical and myopathological findings of nine myositis patients with U1-RNP antibodies who fulfilled the diagnostic criteria for MCTD.</p><p><strong>Results: </strong>Eight patients had muscle weakness in the proximal extremities, and overall, six patients had atypical weakness in the face, neck, wrist, or fingers. Four of those patients required additional intensive treatment (intravenous immunoglobulin or methylprednisolone). Therapeutic responses were consistently favorable overall, and there were no deaths during the observation period. In biopsied muscle specimens, common findings were mild myogenic change, increased necrotic and regenerating fibers, and inflammatory infiltrates predominating in the perivascular region. Two specimens were classified into the spectrum of dermatomyositis (DM); the remaining seven specimens, which had a smaller number of necrotic fibers and nonspecific infiltration, were unclassifiable.</p><p><strong>Discussion: </strong>Patients with MCTD myositis often exhibit an axial or atypical distribution of muscle weakness, which may require intensive therapy. Histological study demonstrates the heterogeneity of myopathology of MCTD myositis and suggests that DM and underlying vasculopathy might be present in these patients.</p>","PeriodicalId":18968,"journal":{"name":"Muscle & Nerve","volume":" ","pages":""},"PeriodicalIF":2.8,"publicationDate":"2025-01-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143059769","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Prophylactic Use of Cardiac Medications and Survival in Duchenne Muscular Dystrophy.","authors":"Kristin M Conway, Shiny Thomas, Tahereh Neyaz, Emma Ciafaloni, Joshua R Mann, Michelle Staron-Ehlinger, Gary S Beasley, Paul A Romitti, Katherine D Mathews","doi":"10.1002/mus.28353","DOIUrl":"https://doi.org/10.1002/mus.28353","url":null,"abstract":"<p><strong>Introduction/aims: </strong>Prophylactic treatment of left ventricular dysfunction (LVD) in Duchenne muscular dystrophy (DMD) delays onset of LVD, but there is limited data showing impact on survival. Our aim was to describe survival among treated and untreated individuals with DMD.</p><p><strong>Methods: </strong>Retrospective, population-based surveillance data from the Muscular Dystrophy Surveillance, Tracking and Research Network (MD STARnet) were used. We analyzed 327 males with DMD born between 1982 and 2009 who were at least 6 years old at the last visit and who initiated cardiac prophylactic medication before age 14 years. Death status was ascertained through vital record linkages and medical record review. Prophylaxis was defined as cardiac medication use at least 1 year before LVD onset (ejection fraction < 55% or shortening fraction < 28%). Age at first visit, corticosteroid use, scoliosis surgery, initiation of noninvasive ventilation, and loss of ambulation were also coded. Cox Proportional Hazard modeling with time-varying covariates describes associations.</p><p><strong>Results: </strong>Prophylactic cardiac treatment was documented for 27.7% (n = 90); corticosteroids were used by 60.9% (n = 157). Adjusting for age at first visit and MD STARnet site, prophylactic treatment was associated with a 54% lower hazard of death (HR = 0.46, 95% CI = 0.22-0.93) compared to no prophylaxis. Adjusting for selected clinical covariates did not appreciably change the estimate (HR = 0.46, 95% CI = 0.22-0.99).</p><p><strong>Discussion: </strong>Initiation of cardiac medication when left ventricular function is normal was associated with prolonged survival in this study of males with DMD. Only one-quarter of individuals received this treatment, however, indicating a topic of focus for improving care.</p>","PeriodicalId":18968,"journal":{"name":"Muscle & Nerve","volume":" ","pages":""},"PeriodicalIF":2.8,"publicationDate":"2025-01-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143033740","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Ultrasound Evaluation of Upper Limb Sublesional Muscle Morphology in Cervical Spinal Cord Injury.","authors":"Hannah J Ro, Emmanuel Ogalo, Mathew I B Debenham, Harvey Wu, Amy K Hanlan, Russell O'Connor, Sean G Bristol, Christopher J Doherty, Erin E Brown, Michael J Berger","doi":"10.1002/mus.28358","DOIUrl":"https://doi.org/10.1002/mus.28358","url":null,"abstract":"<p><strong>Introduction/aims: </strong>Upper limb paralysis is arguably the most limiting consequence of cervical spinal cord injury (cSCI). There is limited knowledge regarding the early structural changes of muscles implicated in grasp/pinch function and upper extremity nerve transfer surgeries. We evaluated: (1) muscle size and echo intensity (EI) in subacute cSCI (2-6 months) and (2) the influence of lower motor neuron (LMN) damage on these ultrasound parameters.</p><p><strong>Methods: </strong>Cross-sectional B-mode images were captured bilaterally in individuals with cSCI (injury duration: 3.3 ± 1.2 months; C4-C6 injury levels; American Spinal Injuries Association Impairment Scale A-C; 45.7 ± 13.7 years; 3 females, 14 males) for biceps brachii (BB), extensor carpi ulnaris, extensor indicis proprius, flexor pollicis longus (FPL), and first dorsal interosseous. Each limb was analyzed as an independent event (n = 34). Cross-sectional area (CSA), thickness (MT), and EI were compared to healthy controls (HC). BB and FPL concentric needle electromyography (EMG) data were also obtained. Abnormal LMN health was defined by the presence of pathological spontaneous activity.</p><p><strong>Results: </strong>Relative to HC, forearm and hand muscle size were 15%-41% lower (p < 0.05), while EI was 21%-40% higher (p < 0.05); no significant differences were observed for sublesional BB muscles (n = 16) (p > 0.05). Muscles demonstrating abnormal LMN health displayed reduced BB MT and elevated FPL EI (p < 0.05).</p><p><strong>Discussion: </strong>These results underscore the substantial changes in forearm and hand muscle morphology within the subacute period after cSCI, with preliminary evidence suggesting that these changes are influenced by LMN damage.</p>","PeriodicalId":18968,"journal":{"name":"Muscle & Nerve","volume":" ","pages":""},"PeriodicalIF":2.8,"publicationDate":"2025-01-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143033743","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Electrophysiological Abnormalities in Finger Extension Weakness and DOwnbeat Nystagmus Motor Neuron Disease: Three New Patients and Review of the Literature.","authors":"Julian Theuriet, Emilien Bernard, Nathalie Guy, Frédéric Taithe, Cécilia Even, Thierry Maisonobe, Aude Sangaré, Pierre Lardeux, Caroline Froment Tilikete, Philippe Couratier, Timothée Lenglet, Antoine Pegat","doi":"10.1002/mus.28357","DOIUrl":"https://doi.org/10.1002/mus.28357","url":null,"abstract":"<p><strong>Introduction/aims: </strong>Finger Extension Weakness and DOwnbeat Nystagmus Motor Neuron Disease (FEWDON-MND) is characterized by motor weakness predominantly affecting finger extension, accompanied by downbeat nystagmus. To date, only 11 patients have been reported. The present study adds a further three and aims to provide a more detailed description of the electrodiagnostic features of these patients.</p><p><strong>Methods: </strong>We present the clinical and electrophysiological features of three French patients from specialized motor neuron centers and review the electrophysiological findings of previously reported patients.</p><p><strong>Results: </strong>These three patients presented with pure motor weakness affecting finger extension and downbeat nystagmus. They exhibited a slowly progressive disease course without respiratory involvement. Nerve conduction studies showed decreased compound muscle action potential amplitudes in the extensor indicis muscles. Abnormal spontaneous activity on needle electromyography (EMG) was rare in two patients, absent in one, and otherwise limited to weak muscles. Additionally, chronic motor axon loss features suggestive of motor neuronopathy were seen in our patients. Importantly, they were also detected in distant asymptomatic muscles.</p><p><strong>Discussion: </strong>The three patients reported here confirm the typical phenotype of FEWDON-MND, characterized by slowly progressive distal motor weakness initially affecting finger extension, associated with downbeat nystagmus. Although chronic motor axon loss features have been found in all reported patients, our three patients show that active denervation can be absent or rare. Thus, finger drop and diffuse chronic neurogenic changes on EMG should lead clinicians to look for downbeat nystagmus and to consider FEWDON-MND.</p>","PeriodicalId":18968,"journal":{"name":"Muscle & Nerve","volume":" ","pages":""},"PeriodicalIF":2.8,"publicationDate":"2025-01-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143024181","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Incidence of Radiologically Confirmed Fractures in Adults With Duchenne Muscular Dystrophy.","authors":"Gavin Langlands, Jennifer McKechnie, Maria E Farrugia, Sze Choong Wong","doi":"10.1002/mus.28355","DOIUrl":"https://doi.org/10.1002/mus.28355","url":null,"abstract":"<p><strong>Introduction/aims: </strong>An increased risk of low trauma fractures is well documented in children and adolescents with duchenne muscular dystrophy (DMD). There is limited evidence regarding the fracture incidence of adults with DMD. The aim of this study was to examine radiologically confirmed fractures in adults with DMD and review bone health monitoring.</p><p><strong>Methods: </strong>This was a retrospective review of all adult males ≥ 16 years with DMD under the care of adult physicians in the West of Scotland (2013-2022). Information regarding fractures, bone health monitoring, and bisphosphonate therapy was collected.</p><p><strong>Results: </strong>Thirty-six men (median age at first visit 18.8 years) with DMD were included. Twelve were taking corticosteroids at first review, and a further 12 had previously been taking corticosteroids that were discontinued in childhood or adolescence. The fracture incidence rate was higher in the corticosteroid group (888.9 per 10,000 person years (95% CI 242.2-2275.9)) than in those not on corticosteroids (156.3 per 10,000 person years (95% CI 32.2-456.6)). Eighteen had lateral spine radiographs for vertebral fracture assessment and 15 had a DXA scan for bone density assessment during the follow-up period.</p><p><strong>Discussion: </strong>The fracture incidence in adult men with DMD is more than double that of UK men aged 18-49 years old, with an even higher incidence in those treated with corticosteroids. Fewer than half of the study population underwent bone monitoring. There is a need for enhanced clinical guidance for the monitoring and management of osteoporosis during transition and throughout adulthood.</p>","PeriodicalId":18968,"journal":{"name":"Muscle & Nerve","volume":" ","pages":""},"PeriodicalIF":2.8,"publicationDate":"2025-01-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143024183","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Thoracic Electric Impedance Tomography Detects Lung Volume Changes in Amyotrophic Lateral Sclerosis.","authors":"Gregory Hansen, Adam Shaw, Kala Bolt, Ryan Verity, Richard T Nataraj, Kerri Lynn Schellenberg","doi":"10.1002/mus.28354","DOIUrl":"https://doi.org/10.1002/mus.28354","url":null,"abstract":"<p><strong>Introduction/aims: </strong>Spirometry is the conventional means to measure lung function in amyotrophic lateral sclerosis (ALS), but is dependent on patient effort and bulbar strength. We aimed to use electric impedance tomography (EIT), an emerging non-invasive imaging modality, to measure dynamic lung volume changes.</p><p><strong>Methods: </strong>Twenty-one patients with ALS underwent sitting and supine spirometry for forced vital capacity (FVC), and sitting and supine EIT. There were 13 patients in the high FVC group (FVC ≥ 80% predicted) and 8 in the low FVC group (FVC < 80% predicted). Additional demographic and clinical data were collected from clinical records.</p><p><strong>Results: </strong>Only the low FVC group had significant loss of lung volumes in the supine position (R² = 0.89 and p < 0.001). The supine volume loss measurement at 10 min correlated with sitting (r² = 0.47) and supine FVC (r² = 0.36), maximum inspiratory (r² = -0.44) and expiratory pressures (r² = 0.36) (MIP and MEP), and the ALS Functional Rating Scale-Revised (ALSFRS-R) dyspnea subscore (r² = 0.36).</p><p><strong>Discussion: </strong>EIT is an emerging alternative to existing measures of lung function in ALS, but without need for patient effort or bulbar strength. Significant losses in lung volume are seen on supine compared to upright position in patients with respiratory dysfunction. Further study is needed to determine relationships to existing clinical measures.</p>","PeriodicalId":18968,"journal":{"name":"Muscle & Nerve","volume":" ","pages":""},"PeriodicalIF":2.8,"publicationDate":"2025-01-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143033742","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Treatment With Full-Spectrum Cannabidiol Oil Improved the Pathological Findings of Dystrophic Mutant Mice.","authors":"Laís Leite Ferreira, Fabricio Souza Gomes, Beatriz Godinho Nascimento, Wagner Corsini, Luis Felipe Cunha Dos Reis, João Marcos Oliveira-Silva, Josie Resende Torres da Silva, Marcelo Lourenço da Silva, Angel Maurício Castro Gamero, Túlio de Almeida Hermes","doi":"10.1002/mus.28337","DOIUrl":"https://doi.org/10.1002/mus.28337","url":null,"abstract":"<p><strong>Introduction/aims: </strong>Duchenne muscular dystrophy (DMD) is caused by pathogenic variants in the DMD gene, making muscle fibers susceptible to contraction-induced membrane damage. Given the potential beneficial action of cannabidiol (CBD), we evaluated the in vitro effect of full-spectrum CBD oil on the viability of dystrophic muscle fibers and the in vivo effect on myopathy of the mdx mouse, a DMD model.</p><p><strong>Methods: </strong>In vitro, dystrophic cells from the mdx mouse were treated with full-spectrum CBD oil and assessed with cell viability and cytotoxic analyses. In vivo, fourteen-day-old mdx mice received 10 mg/kg/day of the full-spectrum CBD oil for 14 days. We analyzed creatine kinase (CK) levels, liver damage markers, and histopathology of the diaphragm (DIA) and quadriceps (QUA [myonecrotic fibers with positive IgG staining, regenerated fibers/central nuclei, the minimum Feret's diameter, the fibrosis area, the inflammatory area, the presence of macrophages, and NF-kappa B content]).</p><p><strong>Results: </strong>In vitro treatment with full-spectrum CBD oil showed a dose-dependent cytotoxic effect; however, in vivo 10 mg/kg treatment was safe and effectively improved DMD histopathological assessment parameters in DIA and QUA: reduction of central nuclei: 1.7% ± 2.0% versus 22.4% ± 5.3% and 11.1% ± 10.7% versus 32.3% ± 4.6%; reduction of IgG+ myofibers: 0.6% ± 0.7% versus 8.4% ± 1.6% and 0.9% ± 0.3% versus 7.5% ± 1.0%; increase in myofiber size: 85.2 ± 3.2 versus 64.3 ± 4.0 μm and 106.5 ± 8.6 versus 81.2 ± 4.8 μm; decrease in inflammatory area: 6.2% ± 2.7% versus 15.1% ± 2.6% and 5.3 ± 4.1 versus 17.3% ± 2.8%; reduced macrophage area: 0.05% ± 0.1% versus 10.8% ± 4.3% and 1.0% ± 0.7% versus 10.3% ± 4.9%; NF-κB levels: 0.6% ± 0.1% versus 1.7% ± 0.2% and 1.7% ± 0.1% versus 5.2% ± 2.1%; and fibrosis: 5.6% ± 1.8% versus 12.0% ± 3.7% and 1.3% ± 0.5% versus 4.7% ± 1.5%. It also reduced serum CK.</p><p><strong>Discussion: </strong>Full-spectrum CBD oil may represent a promising new approach to treating DMD, but its potential toxicity must be considered.</p>","PeriodicalId":18968,"journal":{"name":"Muscle & Nerve","volume":" ","pages":""},"PeriodicalIF":2.8,"publicationDate":"2025-01-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143007658","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}