Maya R Silver, Laurel Caffeé, Lawrence E Fried, Alexander K Gonzalez, Rachael Huber, Michael C Kaufman, Margaret Means, Neil Patel, Mark Ramos, Stephanie Witzman, Katelyn Beam, Jessica Zook, Nicholas S Abend, Craig Press, Sara Molisani
{"title":"A Quality Improvement Initiative to Reduce Underdosing of Rectal Diazepam as Home Seizure Rescue Medication After Hospitalization.","authors":"Maya R Silver, Laurel Caffeé, Lawrence E Fried, Alexander K Gonzalez, Rachael Huber, Michael C Kaufman, Margaret Means, Neil Patel, Mark Ramos, Stephanie Witzman, Katelyn Beam, Jessica Zook, Nicholas S Abend, Craig Press, Sara Molisani","doi":"10.1212/CPJ.0000000000200529","DOIUrl":"10.1212/CPJ.0000000000200529","url":null,"abstract":"<p><strong>Background and objectives: </strong>Seizure rescue medications are commonly prescribed to patients with epilepsy to treat and prevent clusters of seizures or status epilepticus. Underdosing of rescue medications decreases their efficacy, which may lead to status epilepticus and potentially avoidable emergency department (ED) visits or hospitalizations. In this quality improvement initiative, we aimed to reduce the rate of underdosed rectal diazepam prescriptions for children discharged from the inpatient neurology service at our institution from a baseline of 6% to 3% by July 2023.</p><p><strong>Methods: </strong>The primary intervention was an order panel in the electronic health record that automated selection of correct dosing for age-based and weight-based seizure rescue medications including rectal diazepam, intranasal diazepam, and intranasal midazolam. A statistical process control p-chart was used to analyze our primary outcome measure, the monthly rate of underdosed rectal diazepam prescriptions for patients discharged from the inpatient neurology service. The process measure was use of the new order panel. Balancing measures included the dispense rate, cost for seizure rescue medications, and provider satisfaction.</p><p><strong>Results: </strong>During the baseline period, July 2020-August 2022, rectal diazepam was underdosed for 6% of patients discharged from the neurology service. After intervention, we achieved and sustained 0% underdosing of rectal diazepam. We observed no concerning changes in the dispense rate for the medications, the average copay cost remained low, and surveys of ordering providers showed overall high rates of satisfaction. By spreading the intervention, we reduced underdosing from 21% to 0.6% in the ED and from 12% to 4% across the health care system.</p><p><strong>Discussion: </strong>An order panel that automated selection of correct dosing effectively and sustainably reduced underdosing of seizure rescue medications and is transferrable across care settings. We expect that proper dosing of seizure rescue medications should reduce the occurrence of status epilepticus and associated complications.</p>","PeriodicalId":19136,"journal":{"name":"Neurology. Clinical practice","volume":"15 5","pages":"e200529"},"PeriodicalIF":3.2,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12396600/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144962601","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Daniel Karlin, Joanna Smolenski, Daniel Edward Callies, Joseph A Raho
{"title":"Assessing Capacity and Exploring Goals of Care in Locked-In Syndrome: A Clinician's Guide.","authors":"Daniel Karlin, Joanna Smolenski, Daniel Edward Callies, Joseph A Raho","doi":"10.1212/CPJ.0000000000200527","DOIUrl":"10.1212/CPJ.0000000000200527","url":null,"abstract":"<p><p>Patients with locked-in syndrome (LIS) pose unique ethical challenges for decision making, given the complexities of communication. However, some of these patients may retain the ability to participate in decisions regarding their care, including whether to continue life-sustaining treatment or pursue comfort care only. Following a deidentified case, this article has 2 central aims. First, it examines the ethical foundations of decision making and the complexities of capacity assessments for this patient population. Second, it offers a practical guide for neurologists to use when making such evaluations. This guide facilitates a way of communicating with patients with LIS so that clinicians may more systematically assess the patient's capacity to make their own consequential medical decisions.</p>","PeriodicalId":19136,"journal":{"name":"Neurology. Clinical practice","volume":"15 5","pages":"e200527"},"PeriodicalIF":3.2,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12396601/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144962538","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Sara W Hyman, Daniel N de Souza, Laura J Balcer, Steven L Galetta, Laurence R Gore, Jennifer Bickel, Neil A Busis
{"title":"Faculty Perspectives on Appreciation Strategies in a Neurology Department.","authors":"Sara W Hyman, Daniel N de Souza, Laura J Balcer, Steven L Galetta, Laurence R Gore, Jennifer Bickel, Neil A Busis","doi":"10.1212/CPJ.0000000000200523","DOIUrl":"10.1212/CPJ.0000000000200523","url":null,"abstract":"<p><strong>Background and objectives: </strong>Burnout is a pervasive occupational hazard for neurologists-undermining their well-being, jeopardizing patient safety and satisfaction, limiting access to care, and inflating health care costs. Well-designed appreciation and recognition practices may help mitigate some of its key drivers. This pilot study evaluates faculty perspectives on appreciation strategies in an academic neurology department. We used the Moffitt Provider Appreciation Assessment (MPAA), which assesses the types of appreciation methods respondents value, regardless of whether those practices are currently implemented in their workplace.</p><p><strong>Methods: </strong>A cross-sectional survey was conducted among full-time clinical faculty in the Department of Neurology at NYU Grossman School of Medicine. The survey included demographics, the MPAA, the single-item Mini-Z burnout inventory to assess self-reported burnout levels, and an intent-to-leave question. MPAA responses were analyzed for frequencies, and the association between burnout and intent to leave was examined.</p><p><strong>Results: </strong>Of the 202 faculty members, 77 (38%) participated. The most highly valued appreciation practices were inclusion in decision making (77%), efforts to reduce daily frustrations (75%), and positive feedback from patients and families (71%). Gifts, team celebrations with food, and public affirmations were among the least valued practices. Burnout symptoms were reported by 45% of respondents, and 30% indicated an intent to leave within 2 years. Higher burnout levels were associated with increased intent to leave (<i>p</i> < 0.00001). Because the scores for self-reported burnout and intent to leave reflect current work conditions while MPAA scores capture enduring personal values, MPAA rankings cannot be compared directly with burnout or turnover metrics.</p><p><strong>Discussion: </strong>Neurology clinical faculty prioritized appreciation methods that directly address clinical work, underscoring the value of implementing tailored recognition practices that may reduce burnout. The methodology used in this pilot study can be adapted for broader application in other settings. After identifying faculty preferences, health care organizations can implement meaningful, transparent, and inclusive appreciation strategies that have the potential to strengthen physician relationships, promote well-being, and support a sustainable workforce.</p>","PeriodicalId":19136,"journal":{"name":"Neurology. Clinical practice","volume":"15 5","pages":"e200523"},"PeriodicalIF":3.2,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12418805/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145040820","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Jeffrey Buchhalter, Emily Klatte, Zachary M Grinspan, Justin Martello, Michael S Phipps, Lidia Moura
{"title":"An Introduction to Quantitative Aspects of Quality Improvement Science.","authors":"Jeffrey Buchhalter, Emily Klatte, Zachary M Grinspan, Justin Martello, Michael S Phipps, Lidia Moura","doi":"10.1212/CPJ.0000000000200518","DOIUrl":"https://doi.org/10.1212/CPJ.0000000000200518","url":null,"abstract":"<p><p>The use of quality improvement (QI) has become widespread in medicine to improve patient and process outcomes through the methodology of improvement science. Each project follows a format that includes precisely specifying an aim, drivers/barriers to achieving that aim, and potential change concepts/interventions. It is then necessary to define how the data will be collected and displayed and how the significance of the change due to the interventions will be assessed. In this article, we describe the quantitative methods to provide a framework for the performance and reporting of QI projects.</p>","PeriodicalId":19136,"journal":{"name":"Neurology. Clinical practice","volume":"15 5","pages":"e200518"},"PeriodicalIF":3.2,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12342228/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144847963","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Anna Mercante, Harold Siden, Justin Nathaniel Baker, Danai Papadatou, Huda Abu-Saad Huijer, Boris Zernikow, Julie Hauer, Franca Benini
{"title":"Insights From the International Consensus on Neuro-Irritability in Pediatric Palliative Care: Expanding the Known, Challenging the Unknown.","authors":"Anna Mercante, Harold Siden, Justin Nathaniel Baker, Danai Papadatou, Huda Abu-Saad Huijer, Boris Zernikow, Julie Hauer, Franca Benini","doi":"10.1212/CPJ.0000000000200525","DOIUrl":"10.1212/CPJ.0000000000200525","url":null,"abstract":"<p><strong>Background and objectives: </strong>Many children with severe neurologic impairment (SNI) have complex care coordination and management needs, making them eligible for pediatric palliative care (PPC). Indeed, SNI is associated with numerous, often simultaneous, distressing symptoms. Among these, neuro-irritability is particularly common and impactful. The limited understanding of this condition poses a challenge for clinicians in terms of recognition and management, constituting a significant barrier to adequate care. Given the lack of scientific evidence and clinical practice guidelines, we initiated an international and multidisciplinary project to obtain consensus-based guidance on pediatric neuro-irritability.</p><p><strong>Methods: </strong>A panel of 66 experts from across the world was selected to participate in a 2-round Delphi method. The aim of this process was to gather their opinions and insights in the areas of Definition, Assessment, Monitoring, and Treatment of neuro-irritability as encountered in PPC. Panelists were asked to indicate their level of agreement with a series of statements through an online survey, using a 5-point Likert scale. Consensus on a particular item was defined as achieving ≥75% of (dis)agreement among participants.</p><p><strong>Results: </strong>A total of 55 statements were endorsed during the 2 voting sessions. In addition to defining the fundamental features of neuro-irritability, several core elements of the diagnostic process and follow-up protocol were developed. Recommendations on the pharmacological and nonpharmacological approaches are also provided.</p><p><strong>Discussion: </strong>This first international expert consensus aims to support physicians in identifying and addressing neuro-irritability in children with SNI. Our work provides a framework to enhance knowledge on neuro-irritability and advance clinical practice, identifying unmet needs and areas of uncertainty that warrant further research efforts.</p>","PeriodicalId":19136,"journal":{"name":"Neurology. Clinical practice","volume":"15 5","pages":"e200525"},"PeriodicalIF":3.2,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12342229/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144835858","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Jarrett Leech, Brian M Sauer, Amiran Baduashvili, John R Corboy, Thomas Edward Ragole, Stefan Sillau, Russell J Van Coevering, Dana Coutts, Ivan Trang, Kelli M Money, Aaron M Carlson
{"title":"Diagnostic Accuracy of Oligoclonal Bands for CNS Autoimmune Disorders in Acutely Ill Adults.","authors":"Jarrett Leech, Brian M Sauer, Amiran Baduashvili, John R Corboy, Thomas Edward Ragole, Stefan Sillau, Russell J Van Coevering, Dana Coutts, Ivan Trang, Kelli M Money, Aaron M Carlson","doi":"10.1212/CPJ.0000000000200510","DOIUrl":"10.1212/CPJ.0000000000200510","url":null,"abstract":"<p><strong>Background and objectives: </strong>The presence of oligoclonal bands (OCBs) indicates an augmented immune response within the CNS and is integral to the diagnosis of multiple sclerosis (MS). Expert consensus panels recommend OCB testing for conditions other than MS, despite limited data to suggest diagnostic value. Our objectives were to evaluate the spectrum of disease etiologies associated with OCBs and determine the accuracy of OCBs in identifying autoimmune disorders affecting the CNS.</p><p><strong>Methods: </strong>This is a retrospective, observational, single health system study that captured all patients in acute care settings who underwent OCB testing from January 1, 2018, through December 31, 2020. Patient diagnoses and diagnostic categories were adjudicated by clinicians. Test accuracy characteristics to detect CNS autoimmune disorders were computed by comparing 2 or more OCBs as a positive index test against target disease etiologies as reference standards.</p><p><strong>Results: </strong>OCB testing was performed across all etiologies in 926 patients; 78 patients had MS, and 148 had CNS autoimmune disorders not related to MS. Oligoclonal bands exhibited high sensitivity for MS but low sensitivity and low predictive power for other CNS autoimmune disorders.</p><p><strong>Discussion: </strong>In contrast to their diagnostic accuracy in MS, OCBs exhibited limited discriminatory power in the detection of other autoimmune disorders affecting the CNS. Beyond suspected MS, OCBs should not be relied on as an accurate diagnostic tool because a positive result can manifest in an array of neurologic diseases and a negative result does not exclude an autoimmune etiology.</p>","PeriodicalId":19136,"journal":{"name":"Neurology. Clinical practice","volume":"15 5","pages":"e200510"},"PeriodicalIF":3.2,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12413176/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145015897","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"The Leeds Motor Neuron Disease Percutaneous Endoscopic Gastrostomy Pathway: Improving Clinical Outcomes and Experience in a UK Center.","authors":"Linford Fernandes, Priya Devi Shanmugarajah, Zeid Yasiry, Agam Jung","doi":"10.1212/CPJ.0000000000200535","DOIUrl":"10.1212/CPJ.0000000000200535","url":null,"abstract":"<p><strong>Background and objectives: </strong>We outline a quality improvement project on the development of a percutaneous endoscopic gastrostomy (PEG) pathway for patients with motor neuron disease (MND) in a single UK specialist hospital.</p><p><strong>Methods: </strong>Data were collected for 110 patients with MND from 2011 to 2014 before the development of the MND PEG pathway in 2015 and thereafter from 2018 to 2022.</p><p><strong>Results: </strong>Time from MND diagnosis to PEG discussion, time from discussion to referral, and time from referral to PEG insertion were shorter under the new pathway which is of clinical significance, although not statistically significant. The infection rate post-PEG insertion was significantly reduced. Financial savings were made due to the reduced length of stay and no intensive care unit admissions post-PEG insertion.</p><p><strong>Discussion: </strong>Our PEG pathway standardized and streamlined services resulting in reduction of total time taken in the pathway: from initial discussion to decision and procedure. It also reduced post-PEG infections and overall costs.</p>","PeriodicalId":19136,"journal":{"name":"Neurology. Clinical practice","volume":"15 5","pages":"e200535"},"PeriodicalIF":3.2,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12421907/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145040807","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Understanding the Opportunities in Dual Therapy and Emergency Department Utilization in Migraine Care.","authors":"Janine Moore, Alexis Kurek, Kimberly Vo, Kennedy Boone-Sautter, Grace Jipping, Aiesha Ahmed","doi":"10.1212/CPJ.0000000000200519","DOIUrl":"10.1212/CPJ.0000000000200519","url":null,"abstract":"<p><strong>Background and objectives: </strong>Value-based payment (VBP) models require organizations to understand conditions that are contributing to spending and utilization above negotiated targets. Headache was a top 10 reason for an emergency department (ED) visit in 2022. The high utilization continued with more than 2,000 ED visits for migraine in 2023. Nearly all these visits resulted in same-day discharge home, suggesting these may be avoidable. A Payvider council was used to affect medical policy change, and dual therapy for migraine treatment (concurrent use of anti-calcitonin gene-related peptide medications and onabotulinumtoxinA) was approved for coverage in May 2023. This study provides insight into the effect on cost and ED utilization of the medical policy change enabling dual therapy treatment of migraine by comparing cost for pharmacy and ED utilization across monotherapy or dual therapy patient groups.</p><p><strong>Methods: </strong>Patients with a prescription for 1 (monotherapy) or both (dual therapy) medications of interest with an ED encounter for migraine were reviewed. Based on the medications prescribed, patients were classified as receiving monotherapy or dual therapy for the treatment of migraine. The rates of emergency department utilization, ED utilization costs, and pharmacy costs were compared between the groups. This analysis was extrapolated to the larger migraine population within the organization to understand the cost effect of changing migraine management to the organization.</p><p><strong>Results: </strong>Three hundred fifty-seven health plan members used the ED posttreatment between January 1, 2023, and December 31, 2023. A significant difference (<i>p</i> = 0.02) in ED utilization was observed between monotherapy (31.9%, n = 93%) and dual therapy (16.7% n = 11%) patients during the study period. No difference was noted between groups in rate of ED use for patients using the ED. Cost savings associated with ED avoidance, approximately $782 per patient between monotherapy and dual therapy, were insufficient to balance the increased pharmacy cost, which was calculated as approximately $7,115.88 annually per patient based on average wholesale price.</p><p><strong>Discussion: </strong>Dual therapy for migraine had a positive effect on ED visit utilization in our sample while also having cost implications. Health care systems and neurology providers may benefit from this assessment when establishing migraine care, developing data structures, and planning for VBP models.</p>","PeriodicalId":19136,"journal":{"name":"Neurology. Clinical practice","volume":"15 5","pages":"e200519"},"PeriodicalIF":3.2,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12370233/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144962858","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Emily Kutrieb, Montserrat Vera Llonch, Derek Weycker, Steven M Kymes, Duncan Brown, Anne V Smith, Robert S Pulido, Brian Appleby
{"title":"Erratum: Diagnostic Journey and Health Care Burden of Patients With Creutzfeldt-Jakob Disease in the United States: A Real-World Evidence Study.","authors":"Emily Kutrieb, Montserrat Vera Llonch, Derek Weycker, Steven M Kymes, Duncan Brown, Anne V Smith, Robert S Pulido, Brian Appleby","doi":"10.1212/CPJ.0000000000200532","DOIUrl":"10.1212/CPJ.0000000000200532","url":null,"abstract":"<p><p>[This corrects the article DOI: 10.1212/CPJ.0000000000200502.].</p>","PeriodicalId":19136,"journal":{"name":"Neurology. Clinical practice","volume":"15 5","pages":"e200532"},"PeriodicalIF":3.2,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12370232/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144962570","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Matthew P Wicklund, Lindsay N Alfano, Nicholas E Johnson, Peter B Kang, Peter Marks, Katherine D Mathews, Jerry R Mendell, Louise Rodino-Klapac, Douglas Sproule, Nicole Verdun, Kathryn Bryant
{"title":"Limb-Girdle Muscular Dystrophy Scientific Workshop: A Multistakeholder Discussion Focused on Charting the Path Forward for Drug Development.","authors":"Matthew P Wicklund, Lindsay N Alfano, Nicholas E Johnson, Peter B Kang, Peter Marks, Katherine D Mathews, Jerry R Mendell, Louise Rodino-Klapac, Douglas Sproule, Nicole Verdun, Kathryn Bryant","doi":"10.1212/CPJ.0000000000200496","DOIUrl":"https://doi.org/10.1212/CPJ.0000000000200496","url":null,"abstract":"<p><p>Limb-girdle muscular dystrophy (LGMD) refers to a group of muscular dystrophies that generally result in weakness and loss of limb-girdle muscles, leading to severe disability and early mortality due to cardiac and respiratory complications. Heterogeneity across and within individual LGMD subtypes in addition to variability in progression rates presents significant challenges to traditional drug development approaches for these diseases. In an effort to discuss these challenges, as well as opportunities in support of advancing drug development for LGMD, on February 8, 2024, The Speak Foundation assembled a multistakeholder group consisting of academic medical experts, patients and caregivers, patient advocacy organizations, senior leaders from the US Food and Drug Administration, and commercial drug developers. This review will provide an overview of the broad range of topics discussed at the workshop, including LGMD pathophysiology, natural history studies, clinical outcomes, patient-focused drug development, surrogate end points, the Accelerated Approval pathway, and future directions for LGMD drug development.</p>","PeriodicalId":19136,"journal":{"name":"Neurology. Clinical practice","volume":"15 5","pages":"e200496"},"PeriodicalIF":3.2,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12314789/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144775875","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}