Muscle & Nerve最新文献

{"title":"Beyond Prevalence: The Importance of Multifactorial Assessment in Cancer Risk Among Myotonic Dystrophy Patients.","authors":"Christian Messina","doi":"10.1002/mus.70019","DOIUrl":"https://doi.org/10.1002/mus.70019","url":null,"abstract":"","PeriodicalId":18968,"journal":{"name":"Muscle & Nerve","volume":" ","pages":""},"PeriodicalIF":3.1,"publicationDate":"2025-09-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144992934","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":"Atypical Facial Onset Weakness in SOD1 ALS.","authors":"Mark A Garret, Dina Namiranian, Margherita Milone, Arun Varadhachary, Doreen Ho","doi":"10.1002/mus.28428","DOIUrl":"10.1002/mus.28428","url":null,"abstract":"","PeriodicalId":18968,"journal":{"name":"Muscle & Nerve","volume":" ","pages":"520-522"},"PeriodicalIF":3.1,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144034278","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":"Safety Extension Study of Edaravone Oral Suspension in Patients With Amyotrophic Lateral Sclerosis for up to an Additional 96 Weeks of Treatment.","authors":"Angela Genge, Gary L Pattee, Gen Sobue, Masashi Aoki, Hiide Yoshino, Philippe Couratier, Christian Lunetta, Susanne Petri, Daniel Selness, Vesna Todorovic, Nissim Sasson, Manabu Hirai, Fumihiro Takahashi, Alejandro Salah, Stephen Apple, Art Wamil, Alexander Kalin, Carlayne E Jackson","doi":"10.1002/mus.28451","DOIUrl":"10.1002/mus.28451","url":null,"abstract":"<p><strong>Introduction/aims: </strong>Edaravone intravenous (IV) and oral suspension have been shown to have similar pharmacokinetics, safety, and slowing of functional decline in patients with amyotrophic lateral sclerosis (ALS). Study MT-1186-A01 indicated that edaravone oral suspension was well-tolerated over 48 weeks, with no new safety concerns identified relative to existing safety data of IV edaravone, including Study MCI186-19. The aim of this study was to assess the long-term safety and tolerability of edaravone oral suspension in patients with ALS.</p><p><strong>Methods: </strong>Study MT-1186-A03 (NCT04577404) was a phase 3, open-label, multi-center, extension study that evaluated the long-term safety of edaravone oral suspension over an additional 96 weeks in patients with ALS who have completed the initial 48 weeks of Study MT-1186-A01, for a total of up to 144 weeks of treatment. Patients received a 105-mg dose of edaravone administered in treatment cycles identical to the approved edaravone on/off dosing schedule. Patients had definite, probable, probable-laboratory-supported, or possible ALS.</p><p><strong>Results: </strong>In Study MT-1186-A03, edaravone oral suspension was well tolerated with no new safety concerns. The most common treatment-emergent adverse events (TEAEs) were fall, muscular weakness, dyspnea, constipation, and dysphagia. These TEAEs were consistent with the safety profile for edaravone from previous clinical trials.</p><p><strong>Discussion: </strong>These results help establish the long-term safety and tolerability profile of edaravone oral suspension.</p>","PeriodicalId":18968,"journal":{"name":"Muscle & Nerve","volume":" ","pages":"450-454"},"PeriodicalIF":3.1,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12338009/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144248778","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A Systematic Review of Attributes Influencing Preferences for Treatments and Interventions in People With Amyotrophic Lateral Sclerosis (ALS).","authors":"A Clift, D Rowen, L Knox, A W Griffiths, C J McDermott","doi":"10.1002/mus.28437","DOIUrl":"10.1002/mus.28437","url":null,"abstract":"<p><p>Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease that has no cure, and treatments predominantly focus on improving quality of life. Patient-centred care is central to bringing about meaningful improvements to quality of life. This review addresses the lack of consolidated evidence on what matters most to people with ALS (pwALS) by synthesizing 44 preference-based studies covering six different treatment and intervention categories. Data-based convergent synthesis identified five overarching factors influencing preferences: ease of use, accessibility, making life easier, autonomy, and safety/reliability. Simplifying and enhancing accessibility of treatment delivery across disease stages aligns with the nature of neurodegenerative disorders such as ALS, where function declines as the disease progresses. The value in perceived and real control reflects the profound impact ALS has on an individual's independence. Safety and reliability are crucial for people with ALS and are recognized as fundamental requirements for quality healthcare. The themes identified in this review can inform the attributes of preference elicitation methods. Systematically varying the levels of these attributes elicits quantitative measures of preferences. These findings can be used to inform and develop healthcare policy and clinical practice in ALS care. Specifically, preferences related to drug treatments can then be integrated into target product profiles (TPPs) to align drug development with the needs and values of pwALS. Integrating patient preferences into clinical practice promotes patient-centred care, increasing both patient satisfaction and treatment effectiveness.</p>","PeriodicalId":18968,"journal":{"name":"Muscle & Nerve","volume":" ","pages":"359-382"},"PeriodicalIF":3.1,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12338022/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144128233","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Repetitive Nerve Stimulation (RNS) Studies in Infants: Are Infants Just \"Little Adults\" or Do They Deserve Their Own Parameters (or Cut-Off Values) for RNS?","authors":"Sarah E Wright, Tanya J Lehky","doi":"10.1002/mus.28481","DOIUrl":"10.1002/mus.28481","url":null,"abstract":"","PeriodicalId":18968,"journal":{"name":"Muscle & Nerve","volume":" ","pages":"353-355"},"PeriodicalIF":3.1,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144675318","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":"Lymphocyte Activation Gene 3 in Inclusion Body Myositis: A Novel Therapeutic Biomarker?","authors":"Hani Kushlaf","doi":"10.1002/mus.28489","DOIUrl":"10.1002/mus.28489","url":null,"abstract":"","PeriodicalId":18968,"journal":{"name":"Muscle & Nerve","volume":" ","pages":"356-358"},"PeriodicalIF":3.1,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144732370","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":"Multi-Frequency Electrical Impedance Myography as a Predictor of Muscle Function in Older Adults.","authors":"Niladri Kumar Mahato, Buket Sonbas Cobb, Seward B Rutkove, W David Arnold, Brian C Clark","doi":"10.1002/mus.28449","DOIUrl":"10.1002/mus.28449","url":null,"abstract":"<p><strong>Introduction/aims: </strong>This study examined the potential of multi-frequency electrical impedance myography (EIM) to predict hand grip strength (HGS) variability among adults, including a large proportion of older adults, with and without sarcopenia. The objective was to develop predictive models using EIM data for assessing HGS and to explore its potential use in clinical evaluations of muscle function.</p><p><strong>Methods: </strong>Seventy-three participants aged 18-93 years were recruited at community events, with 69 completing EIM and HGS assessments. EIM data were collected from the forearm flexor and extensor muscle groups, and regression techniques, including Lasso regression, were applied to develop predictive models.</p><p><strong>Results: </strong>Approximately 60% of participants were over 65 years old, and 23% exhibited HGS values below sarcopenia thresholds. EIM measurements explained 48% of the variance in HGS. Adding demographic factors (sex, age, BMI) improved the explained variance to 64%, highlighting EIM's potential in muscle health assessments.</p><p><strong>Discussion: </strong>The findings suggest that multi-frequency EIM shows potential as a non-invasive tool for predicting HGS and assessing muscle function, particularly in aging populations. Further research is needed to fully establish EIM's utility in clinical settings for the early detection and management of sarcopenia.</p>","PeriodicalId":18968,"journal":{"name":"Muscle & Nerve","volume":" ","pages":"416-423"},"PeriodicalIF":3.1,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12338028/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144216393","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Pain Experiences and Prescription Pain Medications Among People With Selected Muscular Dystrophies in the Muscular Dystrophy Surveillance, Tracking, and Research Network.","authors":"Jonathan Suhl, Kristin M Conway, Shiny Thomas, Sonja A Rasmussen, James F Howard, Nicholas E Johnson, Paul A Romitti, Katherine D Mathews","doi":"10.1002/mus.28460","DOIUrl":"10.1002/mus.28460","url":null,"abstract":"<p><strong>Introduction/aims: </strong>Pain is a recognized symptom of muscular dystrophy (MD), but little is known about prescription pain medications in this population. We describe pain experiences and pain medications prescribed for individuals with selected MDs using population-based surveillance data collected by the Muscular Dystrophy Surveillance, Tracking, and Research Network.</p><p><strong>Methods: </strong>Pain and prescription data were abstracted from medical records for 1282 individuals with Duchenne and Becker (DBMD) MD during 2000-2015 and congenital (CMD), distal (DD), Emery-Dreifuss (EDMD), facioscapulohumeral (FSHD), limb-girdle (LGMD), and myotonic (DM) MDs during 2008-2016. Percentages of individuals prescribed pain medications for ≥ 6 weeks during follow-up were estimated. Logistic regression was used to examine associations with selected demographic and clinical characteristics.</p><p><strong>Results: </strong>Moderate pain was observed among 34% of all people with available pain scores and varied by MD type (13%-53%). Pain medications were prescribed for 31.1%-40.2% of people 20 years and older, but less frequently (< 15%) among people less than 20 years old. Among people prescribed pain medications, the first medication was typically a non-opioid (57%), but both non-opioid and opioid medication classes were prescribed during follow-up (34%). Pain medications were typically prescribed for longer than 1 year (> 85%). Impaired mobility had the strongest association with prescription pain medication.</p><p><strong>Discussion: </strong>The prescription of pain medication is common for people with symptomatic MD. Most people were prescribed only non-opioids. These data highlight pain management as a frequent component of MD care. Understanding modifiable factors associated with MD-related pain and effective interventions may help improve care.</p>","PeriodicalId":18968,"journal":{"name":"Muscle & Nerve","volume":" ","pages":"475-484"},"PeriodicalIF":3.1,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12306532/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144369087","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Immune Characterization of a Vietnamese Cohort With Idiopathic Inflammatory Myopathies.","authors":"Yue-Bei Luo, Maho Nakazawa, Nham Pham Thi Minh, Léo Heinon, Inger Nennesmo, Johan Rönnelid, Ingrid E Lundberg, Thuy Nguyen Thi Phuong, Begum Horuluoglu","doi":"10.1002/mus.28455","DOIUrl":"10.1002/mus.28455","url":null,"abstract":"<p><strong>Introduction/aims: </strong>Idiopathic inflammatory myopathies (IIMs) are classified into five subtypes that are associated with distinct groups of myositis-specific antibodies (MSAs). Variations in the prevalence, genetic predisposition and clinical manifestations exist in patients with IIM from different ethnic backgrounds. In this study, we aimed to characterize the immunopathological changes in muscle tissue and serum cytokines in a Vietnamese IIM cohort with Kinh ethnicity.</p><p><strong>Methods: </strong>Muscle biopsies, sera, clinical data, and autoantibody profiles from 64 patients with IIM were included. Muscles were stained by immunohistochemistry using antibodies that target immune cells and molecules. Serum cytokines were measured by multiplex bead arrays. Clustering analysis was performed using the partitioning around medoids algorithm.</p><p><strong>Results: </strong>The anti-MDA5 antibody was the most common MSA in this cohort (15.6%), followed by anti-Jo-1 (10.9%). A wide range of clinical manifestations and immunohistological features were seen within the serologically defined subgroups. There was no difference in the levels of immune cells, capillary density or dilation, or patterns of C5b-9 expression between the myositis subgroups. MHC-I patterns were the only immunomorphological variable that was different in these subgroups. IP-10, IL-17A, and APRIL were increased in patients with IIM compared to healthy controls (p = 5.7 × 10^-3, 6.9 × 10^-4, 2.4 × 10^-3, respectively) but without difference between IIM subgroups.</p><p><strong>Discussion: </strong>Vietnamese patients with IIM demonstrated widely varying clinical and immunopathological features, with dermatomyositis and immune-mediated necrotizing myopathy representing opposite ends of MHC-I upregulation in muscle tissue. Thus classification of myositis requires comprehensive evaluation of clinical manifestations, serology profiling and muscle pathology.</p>","PeriodicalId":18968,"journal":{"name":"Muscle & Nerve","volume":" ","pages":"464-474"},"PeriodicalIF":3.1,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12338015/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144302561","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Efficacy and Safety of Once Daily Dosing vs. Approved On/Off Dosing of Edaravone Oral Suspension Up to 48 Weeks in Patients With Amyotrophic Lateral Sclerosis (Study MT-1186-A02).","authors":"Jeffrey Rothstein, Angela Genge, Shari De Silva, Lorne Zinman, Marvin Chum, Adriano Chio, Gen Sobue, Masashi Aoki, Hiide Yoshino, Manabu Doyu, Daniel Selness, Vesna Todorovic, Nissim Sasson, Manabu Hirai, Fumihiro Takahashi, Alejandro Salah, Art Wamil, Stephen Apple","doi":"10.1002/mus.28448","DOIUrl":"10.1002/mus.28448","url":null,"abstract":"<p><strong>Introduction/aims: </strong>An On/Off dosing regimen of intravenous (IV) edaravone and edaravone oral suspension is approved in the US for the treatment of amyotrophic lateral sclerosis (ALS). Placebo-controlled clinical trials showed IV edaravone slows the rate of physical functional decline. This study evaluated whether investigational daily dosing displayed superior efficacy vs. approved on/off dosing of edaravone oral suspension, and assessed safety and tolerability, over 48 weeks in patients with ALS.</p><p><strong>Methods: </strong>Study MT-1186-A02 (NCT04569084) was a multicenter, phase 3b, double-blind, parallel group, superiority study that randomized patients to edaravone oral suspension (105-mg dose) administered Once Daily or the same edaravone oral suspension dose administered according to the approved On/Off regimen including placebo to mimic daily drug dosing. Patients had definite or probable ALS, baseline forced vital capacity ≥ 70%, and baseline disease duration ≤ 2 years. The primary endpoint was a combined assessment of function and survival (CAFS) at week 48, which included change in ALS Functional Rating Scale-Revised (ALSFRS-R) and time to death.</p><p><strong>Results: </strong>CAFS at week 48 indicated Once Daily dosing did not show a statistically significant difference vs. approved on/off dosing (p = 0.777). Both dosing regimens provided comparable change from baseline ALSFRS-R total score to week 48 (least squares mean difference: 0.27 [95% CI -1.43 to 1.97]). Edaravone oral suspension was well tolerated, and no new safety concerns were identified in either group.</p><p><strong>Discussion: </strong>Daily edaravone oral suspension did not show superiority and had equivalent safety and tolerability vs. the approved On/Off regimen, reinforcing the appropriateness of the approved dosing regimen.</p>","PeriodicalId":18968,"journal":{"name":"Muscle & Nerve","volume":" ","pages":"433-442"},"PeriodicalIF":3.1,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12338024/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144234586","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}