Evidence map›Paper›PMID 38769202›Full record

ReviewNature reviews. Neurology2024

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

Michael Benatar, Joanne Wuu, Edward D Huey, Corey T McMillan, Ronald C Petersen, Ronald Postuma, Caroline McHutchison, Laynie Dratch, Jalayne J Arias, Anita Crawley and 17 more

Erratum issuedAbstract readReview
In one paragraph

Review in Nature reviews. Neurology, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 29 papers.

0numbers the graph read from it
0cells of the map it votes in
29citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

29 citing papers in PubMed.

  1. Observational
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  3. Review
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  5. Atypical features including acquired oculomotor apraxia inJournal of neuromuscular diseases · 2026
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4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

27 authors.

Michael BenatarDepartment of Neurology, Miller School of Medicine, University of Miami, Miami, FL, USA. mbenatar@miami.edu.ORCID 0000-0003-4241-5135
Joanne WuuDepartment of Neurology, Miller School of Medicine, University of Miami, Miami, FL, USA.ORCID 0009-0005-9643-9855
Edward D HueyDepartment of Psychiatry and Human Behaviour, Alpert Medical School of Brown University, Providence, RI, USA.
Corey T McMillanDepartment of Neurology, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA.
Ronald C PetersenDepartment of Neurology, Mayo Clinic, Rochester, MN, USA.
Ronald PostumaDepartment of Neurology, Montreal Neurological Institute, McGill University, Montreal, Quebec, Canada.
Caroline McHutchisonHuman Cognitive Neuroscience, Department of Psychology, University of Edinburgh, Edinburgh, UK.
Laynie DratchDepartment of Neurology, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA, USA.ORCID 0000-0002-2179-3199
Jalayne J AriasDepartment of Health Policy & Behavioral Sciences, School of Public Health, Georgia State University, Atlanta, GA, USA.
Anita Crawley, Buffalo, NY, USA.
Henry HouldenUCL Institute of Neurology and The National Hospital for Neurology and Neurosurgery, London, UK.ORCID 0000-0002-2866-7777
Michael P McDermottDepartment of Biostatistics and Computational Biology, University of Rochester School of Medicine and Dentistry, Rochester, NY, USA.
Xueya CaiDepartment of Biostatistics and Computational Biology, University of Rochester School of Medicine and Dentistry, Rochester, NY, USA.
Neil ThakurALS Association, Arlington, VA, USA.ORCID 0000-0003-2099-3364
Adam BoxerDepartment of Neurology, University of California, San Francisco, CA, USA.ORCID 0000-0002-1215-5064
Howard RosenDepartment of Neurology, University of California, San Francisco, CA, USA.
Bradley F BoeveDepartment of Neurology, Mayo Clinic, Rochester, MN, USA.ORCID 0000-0002-4153-8187
Penny DacksAssociation for Frontotemporal Degeneration, King of Prussia, PA, USA.
Stephanie CosentinoDepartment of Psychiatry, Columbia University, New York, NY, USA.
Sharon AbrahamsHuman Cognitive Neuroscience, Department of Psychology, University of Edinburgh, Edinburgh, UK.
Neil ShneiderDepartment of Neurology, Columbia University, New York, NY, USA.ORCID 0000-0002-3223-7366
Paul LingorDepartment of Neurology, Klinikum rechts der Isar der Technischen Universität München, Munich, Germany.ORCID 0000-0001-9362-7096
Jeremy ShefnerDepartment of Neurology, Barrow Neurological Institute, Phoenix, AZ, USA.
Peter M AndersenDepartment of Clinical Science, Neurosciences, Umeå University, Umeå, Sweden.ORCID 0000-0003-0094-5429
Ammar Al-ChalabiDepartment of Basic and Clinical Neuroscience, Maurice Wohl Clinical Neuroscience Institute, King's College London, London, UK.ORCID 0000-0002-4924-7712
Martin R TurnerNuffield Department of Clinical Neurosciences, University of Oxford, Oxford, UK.ORCID 0000-0003-0267-3180
Attendees of the Second International Pre-Symptomatic ALS Workshop

Funding

Uncovering new genes and disease modifiers for ALS and related disordersU54NS092091 · NINDS · UNIVERSITY OF MIAMI SCHOOL OF MEDICINE · PI Michael Benatar · 2014 to 2026
$19.8M
Pre-Symptomatic Familial ALS (Pre-fALS) Study: From Prodrome and Biomarkers to ALS/FTD PreventionRF1NS105479 · NINDS · UNIVERSITY OF MIAMI SCHOOL OF MEDICINE · PI BENATAR, MICHAEL, WUU, JOANNE · 2025 to 2025
$11.3M
Genetic basis of neuropsychiatric symptoms in Alzheimer's diseaseU01AG079850 · NIA · COLUMBIA UNIVERSITY HEALTH SCIENCES · PI Gary Wayne Beecham, Edward D Huey · 2023 to 2026
$3.0M
Pre-Symptomatic Familial ALS (Pre-fALS) Study - Prelude to a Treatment Trial; Administrative SupplementR01NS105479 · NINDS · UNIVERSITY OF MIAMI SCHOOL OF MEDICINE · PI BENATAR, MICHAEL, WUU, JOANNE · 2018 to 2022
$3.0M
Neuroanatomical associations with the factor structure underlying neuropsychiatric symptoms in Alzheimer's diseaseR01AG062268 · NIA · COLUMBIA UNIVERSITY HEALTH SCIENCES · PI HUEY, EDWARD D · 2018 to 2022
$2.5M
Using RDoC Negative and Positive Valence Paradigms to Investigate the Mechanisms of Neuropsychiatric Symptoms (NPS) in Alzheimer's Disease and Related DementiasR01MH120794 · NIMH · COLUMBIA UNIVERSITY HEALTH SCIENCES · PI HUEY, EDWARD D · 2019 to 2023
$2.1M
Employment and Insurance Discrimination Based on Biomarkers for Alzheimer's diseaseK01AG057796 · NIA · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI ARIAS, JALAYNE J · 2018 to 2022
$652k
NIA NIH HHS K01 AG057796NIA NIH HHS R01 AG062268NIA NIH HHS U01 AG079850NIMH NIH HHS R01 MH120794NINDS NIH HHS R01 NS105479NINDS NIH HHS RF1 NS105479NINDS NIH HHS U54 NS092091
6 · The paper itself

Abstract

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.

Indexed as

Amyotrophic Lateral SclerosisFrontotemporal DementiaPhenotypeBiomarkersHumansNeurodegenerative DiseasesBiomarkers

Identifiers

PMID38769202
PMCPMC11216694

What OpenQuestion holds

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Registered trials

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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.