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The Miami Framework for ALS and related neurodegenerative disorders : an integrated view of phenotype and biology

Benatar, Michael (författare)
Department of Neurology, Miller School of Medicine, University of Miami, FL, Miami, United States
Wuu, Joanne (författare)
Department of Neurology, Miller School of Medicine, University of Miami, FL, Miami, United States
Huey, Edward D. (författare)
Department of Psychiatry and Human Behaviour, Alpert Medical School of Brown University, RI, Providence, United States
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McMillan, Corey T. (författare)
Department of Neurology, University of Pennsylvania Perelman School of Medicine, PA, Philadelphia, United States
Petersen, Ronald C. (författare)
Department of Neurology, Mayo Clinic, MN, Rochester, United States
Postuma, Ronald (författare)
Department of Neurology, Montreal Neurological Institute, McGill University, QC, Montreal, Canada
McHutchison, Caroline (författare)
Human Cognitive Neuroscience, Department of Psychology, University of Edinburgh, Edinburgh, United Kingdom; Euan MacDonald Centre for MND Research, University of Edinburgh, Edinburgh, United Kingdom
Dratch, Laynie (författare)
Department of Neurology, University of Pennsylvania Perelman School of Medicine, PA, Philadelphia, United States
Arias, Jalayne J. (författare)
Department of Health Policy & Behavioral Sciences, School of Public Health, Georgia State University, GA, Atlanta, United States
Crawley, Anita (författare)
NY, Buffalo, United States
Houlden, Henry (författare)
UCL Institute of Neurology and The National Hospital for Neurology and Neurosurgery, London, United Kingdom
McDermott, Michael P. (författare)
Department of Biostatistics and Computational Biology, University of Rochester School of Medicine and Dentistry, NY, Rochester, United States; Department of Neurology, University of Rochester School of Medicine and Dentistry, NY, Rochester, United States
Cai, Xueya (författare)
Department of Biostatistics and Computational Biology, University of Rochester School of Medicine and Dentistry, NY, Rochester, United States
Thakur, Neil (författare)
ALS Association, VA, Arlington, United States
Boxer, Adam (författare)
Department of Neurology, University of California, CA, San Francisco, United States
Rosen, Howard (författare)
Department of Neurology, University of California, CA, San Francisco, United States
Boeve, Bradley F. (författare)
Department of Neurology, Mayo Clinic, MN, Rochester, United States
Dacks, Penny (författare)
Association for Frontotemporal Degeneration, PA, King of Prussia, United States
Cosentino, Stephanie (författare)
Department of Psychiatry, Columbia University, New York, NY, USA
Abrahams, Sharon (författare)
Human Cognitive Neuroscience, Department of Psychology, University of Edinburgh, Edinburgh, United Kingdom; Euan MacDonald Centre for MND Research, University of Edinburgh, Edinburgh, United Kingdom
Shneider, Neil (författare)
Department of Neurology, Columbia University, New York, NY, USA
Lingor, Paul (författare)
Department of Neurology, Klinikum rechts der Isar der Technischen Universität München, Munich, Germany
Shefner, Jeremy (författare)
Department of Neurology, Barrow Neurological Institute, AZ, Phoenix, United States
Andersen, Peter M., 1962- (författare)
Umeå universitet,Neurovetenskaper
Al-Chalabi, Ammar (författare)
Department of Basic and Clinical Neuroscience, Maurice Wohl Clinical Neuroscience Institute, King's College London, London, United Kingdom; Department of Neurology, King's College Hospital, London, United Kingdom
Turner, Martin R. (författare)
Nuffield Department of Clinical Neurosciences, University of Oxford, Oxford, United Kingdom
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 (creator_code:org_t)
Springer Nature, 2024
2024
Engelska.
Ingår i: Nature Reviews Neurology. - : Springer Nature. - 1759-4758 .- 1759-4766. ; 20:6, s. 364-376
Relaterad länk:
https://urn.kb.se/re...
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https://doi.org/10.1...
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  • Forskningsöversikt (refereegranskat)
Abstract Ämnesord
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  • Increasing appreciation of the phenotypic and biological overlap between amyotrophic lateral sclerosis (ALS) and frontotemporal dementia, alongside evolving biomarker evidence for a pre-symptomatic stage of disease and observations that this stage of disease might not always be clinically silent, is challenging traditional views of these disorders. These advances have highlighted the need to adapt ingrained notions of these clinical syndromes to include both the full phenotypic continuum - from clinically silent, to prodromal, to clinically manifest - and the expanded phenotypic spectrum that includes ALS, frontotemporal dementia and some movement disorders. The updated clinical paradigms should also align with our understanding of the biology of these disorders, reflected in measurable biomarkers. The Miami Framework, emerging from discussions at the Second International Pre-Symptomatic ALS Workshop in Miami (February 2023; a full list of attendees and their affiliations appears in the Supplementary Information) proposes a classification system built on: first, three parallel phenotypic axes - motor neuron, frontotemporal and extrapyramidal - rather than the unitary approach of combining all phenotypic elements into a single clinical entity; and second, biomarkers that reflect different aspects of the underlying pathology and biology of neurodegeneration. This framework decouples clinical syndromes from biomarker evidence of disease and builds on experiences from other neurodegenerative diseases to offer a unified approach to specifying the pleiotropic clinical manifestations of disease and describing the trajectory of emergent biomarkers.

Ämnesord

MEDICIN OCH HÄLSOVETENSKAP  -- Klinisk medicin -- Neurologi (hsv//swe)
MEDICAL AND HEALTH SCIENCES  -- Clinical Medicine -- Neurology (hsv//eng)

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