{"title":"Prognostic plasma biomarkers for spinal cord injury","authors":"Heather Wood","doi":"10.1038/s41582-025-01075-w","DOIUrl":"10.1038/s41582-025-01075-w","url":null,"abstract":"Measurements of neurofilament light chain, glial fibrillary acidic protein and contactin 1 in plasma can provide useful prognostic information following traumatic spinal cord injury, according to new research.","PeriodicalId":19085,"journal":{"name":"Nature Reviews Neurology","volume":"21 4","pages":"173-173"},"PeriodicalIF":28.2,"publicationDate":"2025-03-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143582805","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Spinal muscular atrophy treatment in utero","authors":"Heather Wood","doi":"10.1038/s41582-025-01076-9","DOIUrl":"10.1038/s41582-025-01076-9","url":null,"abstract":"A recent article reports the successful prenatal treatment of a child with spinal muscular atrophy type 1.","PeriodicalId":19085,"journal":{"name":"Nature Reviews Neurology","volume":"21 4","pages":"173-173"},"PeriodicalIF":28.2,"publicationDate":"2025-03-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143582748","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Elisabetta Indelicato, Martin B. Delatycki, Jennifer Farmer, Marcondes C. França Jr, Susan Perlman, Myriam Rai, Sylvia Boesch
{"title":"A global perspective on research advances and future challenges in Friedreich ataxia","authors":"Elisabetta Indelicato, Martin B. Delatycki, Jennifer Farmer, Marcondes C. França Jr, Susan Perlman, Myriam Rai, Sylvia Boesch","doi":"10.1038/s41582-025-01065-y","DOIUrl":"10.1038/s41582-025-01065-y","url":null,"abstract":"Friedreich ataxia (FRDA) is a rare multisystem, life-limiting disease and is the most common early-onset inherited ataxia in populations of European, Arab and Indian descent. In recent years, substantial progress has been made in dissecting the pathogenesis and natural history of FRDA, and several clinical trials have been initiated. A particularly notable recent achievement was the approval of the nuclear factor erythroid 2-related factor 2 activator omaveloxolone as the first disease-specific therapy for FRDA. In light of these developments, we review milestones in FRDA translational and clinical research over the past 10 years, as well as the various therapeutic strategies currently in the pipeline. We also consider the lessons that have been learned from failed trials and other setbacks. We conclude by presenting a global roadmap for future research, as outlined by the recently established Friedreich’s Ataxia Global Clinical Consortium, which covers North and South America, Europe, India, Australia and New Zealand. Friedreich ataxia (FRDA) is a rare multisystem disorder and is the most common early-onset hereditary ataxia in populations of European, Arab and Indian descent. This article reviews the milestones in FRDA translational and clinical research over the past 10 years and outlines the priorities for future FRDA research.","PeriodicalId":19085,"journal":{"name":"Nature Reviews Neurology","volume":"21 4","pages":"204-215"},"PeriodicalIF":28.2,"publicationDate":"2025-03-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143532343","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Parkinson disease pathology can originate in the kidney","authors":"Lisa Kiani","doi":"10.1038/s41582-025-01066-x","DOIUrl":"10.1038/s41582-025-01066-x","url":null,"abstract":"New research suggests that pathological α-synuclein in Parkinson disease can propagate from kidney to brain.","PeriodicalId":19085,"journal":{"name":"Nature Reviews Neurology","volume":"21 3","pages":"125-125"},"PeriodicalIF":28.2,"publicationDate":"2025-02-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143434932","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Ruth Dobson, Charlotte Kenten, Joanna Brown, Clarissa Giebel, Sube Banerjee, Claudia Cooper
{"title":"Moving towards meaningful patient and public engagement","authors":"Ruth Dobson, Charlotte Kenten, Joanna Brown, Clarissa Giebel, Sube Banerjee, Claudia Cooper","doi":"10.1038/s41582-025-01061-2","DOIUrl":"10.1038/s41582-025-01061-2","url":null,"abstract":"Patient and public involvement and engagement is increasingly mandated in funding applications, yet often remain tokenistic and transitory. Working with patient and public contributors requires investment, thought, care and time. We discuss approaches that aim to increase agency for coresearchers, with the goal of strengthening public confidence and trust in research.","PeriodicalId":19085,"journal":{"name":"Nature Reviews Neurology","volume":"21 3","pages":"121-122"},"PeriodicalIF":28.2,"publicationDate":"2025-02-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143375654","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Nothing about us, without us — establishing a patient and public involvement and engagement group","authors":"Rachel Horne, Rosemary Phillips, Mohammed A. Rauf","doi":"10.1038/s41582-025-01063-0","DOIUrl":"10.1038/s41582-025-01063-0","url":null,"abstract":"Implementation of patient and public involvement and engagement (PPIE) to enable patients and carers to feel included and equal to healthcare professionals is challenging to do well. Here, leaders of a PPIE group share their lived experience and highlight the importance of addressing the needs of all participants to enable true partnership.","PeriodicalId":19085,"journal":{"name":"Nature Reviews Neurology","volume":"21 3","pages":"123-124"},"PeriodicalIF":28.2,"publicationDate":"2025-02-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143375653","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
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