{"title":"The artist and the scientist join forces for rare diseases","authors":"Jules Morgan","doi":"10.1016/s1474-4422(25)00038-9","DOIUrl":"https://doi.org/10.1016/s1474-4422(25)00038-9","url":null,"abstract":"No Abstract","PeriodicalId":22676,"journal":{"name":"The Lancet Neurology","volume":"88 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-03-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143660507","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}
Michele Tinazzi, Marialuisa Gandolfi, Carlo Alberto Artusi, Kirsty Bannister, Katarina Rukavina, Christine Brefel-Courbon, Daniel Ciampi de Andrade, Santiago Perez-Lloret, Veit Mylius
{"title":"Advances in diagnosis, classification, and management of pain in Parkinson's disease","authors":"Michele Tinazzi, Marialuisa Gandolfi, Carlo Alberto Artusi, Kirsty Bannister, Katarina Rukavina, Christine Brefel-Courbon, Daniel Ciampi de Andrade, Santiago Perez-Lloret, Veit Mylius","doi":"10.1016/s1474-4422(25)00033-x","DOIUrl":"https://doi.org/10.1016/s1474-4422(25)00033-x","url":null,"abstract":"With over 10 million people affected worldwide, Parkinson's disease is the fastest-growing neurological disorder. More than two-thirds of people with Parkinson's disease live with chronic pain, which can manifest in various stages of the disease, substantially affecting daily activities and quality of life. The Parkinson's disease Pain Classification System overcomes the limitations of previous classification systems by distinguishing between pain related to Parkinson's disease and unrelated pain, while also incorporating clinical and pathophysiological (mechanistic) descriptors such as nociceptive, neuropathic, and nociplastic pain. This system provides a framework for accurate diagnosis and mechanism-based therapy. Alongside the appropriate classification of pain, consideration of treatment approaches that include non-invasive (pharmacological and non-pharmacological) and invasive strategies tailored to specific types of pain will refine and inform research trials and clinical practice when it comes to treating pain in Parkinson's disease.","PeriodicalId":22676,"journal":{"name":"The Lancet Neurology","volume":"40 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-03-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143660308","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}
Issam A Awad, Roberto J Alcazar-Felix, Agnieszka Stadnik, Serena Kinkade, Aditya Jhaveri, Justine Lee, Stephanie Hage, Javed Iqbal, Sean P Polster, Robert Shenkar, Kevin Treine, Nichol McBee, Noeleen Ostapkovich, Karen Lane, James K Liao, Matthew Sorrentino, Cornelia Lee, Kelly D Flemming, Romuald Girard, Timothy J Carroll, Daniel F Hanley
{"title":"Safety and efficacy of atorvastatin for rebleeding in cerebral cavernous malformations (AT CASH EPOC): a phase 1/2a, randomised placebo-controlled trial","authors":"Issam A Awad, Roberto J Alcazar-Felix, Agnieszka Stadnik, Serena Kinkade, Aditya Jhaveri, Justine Lee, Stephanie Hage, Javed Iqbal, Sean P Polster, Robert Shenkar, Kevin Treine, Nichol McBee, Noeleen Ostapkovich, Karen Lane, James K Liao, Matthew Sorrentino, Cornelia Lee, Kelly D Flemming, Romuald Girard, Timothy J Carroll, Daniel F Hanley","doi":"10.1016/s1474-4422(25)00036-5","DOIUrl":"https://doi.org/10.1016/s1474-4422(25)00036-5","url":null,"abstract":"<h3>Background</h3>Cerebral cavernous malformations (CCMs) carry a high risk of rebleeding after symptomatic haemorrhage, with serious clinical sequelae. Atorvastatin was shown to prevent CCM growth and bleeding in animal models. We aimed to assess the safety and efficacy of atorvastatin on rebleeding in patients with CCMs after a symptomatic haemorrhage.<h3>Methods</h3>We did a phase 1/2a randomised trial at the University of Chicago's CCM Center of Excellence. Patients aged 18–80 years with untreated CCMs who had had symptomatic bleeding from a CCM lesion within the previous year were eligible. Patients were randomly allocated (1:1) to oral atorvastatin (80 mg daily for 2 years) or matching placebo. Investigators, clinical staff, and participants were masked to the assigned treatment. The primary efficacy outcome was the percentage change in mean lesional iron deposition per year, measured by quantitative susceptibility mapping (QSM) on MRI and averaged over 2 years; a decrease would signal potential benefit and an increase a safety concern. The primary efficacy outcome was analysed in the modified intention-to-treat cohort, including patients with at least one annual paired QSM assessment. Safety outcomes included rates of bleeds and serious adverse events necessitating drug discontinuation. This trial is registered at ClinicalTrials.gov (NCT02603328) and is completed.<h3>Findings</h3>Between July 25, 2018, and July 22, 2022, 326 patients were assessed for eligibility, and 80 patients were allocated either atorvastatin (n=41) or placebo (n=39). 29 (36%) patients were male and 51 (64%) were female. 64 (80%) patients (33 in the atorvastatin group and 31 in the placebo group) had at least one annual paired QSM assessment and were included in the modified intention-to-treat analyses. The mean annual percentage change in lesional QSM was 10·88 (SE 7·29) with atorvastatin versus 12·09 (SE 7·54) with placebo (treatment effect –1·22, 95% CI –22·25 to 19·81; p=0·91). Symptomatic haemorrhage was reported in six patients assigned atorvastatin and seven patients assigned placebo (relative risk 0·81, 95% CI 0·31 to 2·13). No patients had a serious adverse event requiring drug discontinuation and no deaths were recorded.<h3>Interpretation</h3>For people with symptomatic haemorrhage caused by CCMs, atorvastatin did not affect the mean change in lesional iron deposition on brain MRI over 2 years when compared with placebo. Atorvastatin was well tolerated and no safety concerns were noted. The study provides a useful framework for biomarker driven drug assessment in a rare disease.<h3>Funding</h3>US National Institutes of Health.","PeriodicalId":22676,"journal":{"name":"The Lancet Neurology","volume":"1 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-03-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143660974","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":"Advocating for increased awareness and research on Down syndrome","authors":"Marie-Claude Potier","doi":"10.1016/s1474-4422(25)00070-5","DOIUrl":"https://doi.org/10.1016/s1474-4422(25)00070-5","url":null,"abstract":"No Abstract","PeriodicalId":22676,"journal":{"name":"The Lancet Neurology","volume":"20 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-03-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143660556","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}
Michael J Young, Christof Koch, Jan Claassen, David Fischer, Charlène Aubinet, Olivia Gosseries, Daniel Kondziella, Mario Rosanova, Simone Sarasso, Silvia Casarotto, Marcello Massimini, Brian L Edlow
{"title":"An ethical framework to assess covert consciousness","authors":"Michael J Young, Christof Koch, Jan Claassen, David Fischer, Charlène Aubinet, Olivia Gosseries, Daniel Kondziella, Mario Rosanova, Simone Sarasso, Silvia Casarotto, Marcello Massimini, Brian L Edlow","doi":"10.1016/s1474-4422(24)00432-0","DOIUrl":"https://doi.org/10.1016/s1474-4422(24)00432-0","url":null,"abstract":"No Abstract","PeriodicalId":22676,"journal":{"name":"The Lancet Neurology","volume":"29 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2025-02-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143451483","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}
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