ArticleNeurology2019
Revised Airlie House consensus guidelines for design and implementation of ALS clinical trials.
Open access · bronzeAbstract readConsensus Statement
In one paragraph
Article in Neurology, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 76 papers, 5 of them syntheses that pooled it.
0numbers the graph read from it
0cells of the map it votes in
76citing papers in PubMed, 5 pooled it
15.2field-weighted citation impact, top 1% of its field
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.
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.
Who cites it
76 citing papers in PubMed, 5 syntheses or guidelines pooled it, 134 citations in OpenAlex.
- Pooled it
- Pooled it
- The landscape of neurophysiological outcome measures in ALS interventional trials: A systematic review.Clinical neurophysiology : official journal of the International Federation of Clinical Neurophysiology · 2022Pooled it
- A systematic review of non-motor symptom evaluation in clinical trials for amyotrophic lateral sclerosis.Journal of neurology · 2022Pooled it
- A systematic review of neuropsychiatric and cognitive assessments used in clinical trials for amyotrophic lateral sclerosis.Journal of neurology · 2021Pooled it
- Add-on treatment with Cerebrolysin improves clinical symptoms in patients with ALS: results from a prospective, single-center, placebo-controlled, randomized, double-blind, phase II study.Journal of medicine and life · 2023Trial
- Trial
- The unfolded protein response in amyotrophic later sclerosis: results of a phase 2 trial.Brain : a journal of neurology · 2021Trial
- Umbilical Cord Mesenchymal Stem Cells in Amyotrophic Lateral Sclerosis: an Original Study.Stem cell reviews and reports · 2020Trial
- Trial of Sodium Phenylbutyrate-Taurursodiol for Amyotrophic Lateral Sclerosis.The New England journal of medicine · 2020Trial
- Repeated 5-day cycles of low dose aldesleukin in amyotrophic lateral sclerosis (IMODALS): A phase 2a randomised, double-blind, placebo-controlled trial.EBioMedicine · 2020Trial
- Powering Neurology Drug Development Trials to Reduce False Negative Results.Clinical and translational science · 2026Review
- The role of adiponectin and cytokines in Amyotrophic lateral sclerosis: assessment of disease progression and survival status.Scientific reports · 2026Article
- Next-Generation Phospholipid Nanocomplexes for Precision Neurotherapeutics: Harnessing Endogenous Blood-Brain Barrier Transport Mechanisms to Revolutionize the Treatment of Neurodegenerative Diseases.Recent advances in drug delivery and formulation · 2026Review
- Traditional Chinese botanical medicines and plant-derived bioactive compounds in amyotrophic lateral sclerosis: mechanistic evidence, translational challenges, and therapeutic opportunities.Frontiers in pharmacology · 2026Review
- Entering the era of precision medicine to treat amyotrophic lateral sclerosis.Molecular neurodegeneration · 2025Review
- Remote Monitoring of Amyotrophic Lateral Sclerosis Using Digital Health Technologies: Shifting Toward Digitalized Care and Research?Neurology · 2025Review
- miRNA-214 to predict progression and survival in ALS.Journal of neurology, neurosurgery, and psychiatry · 2025Article
- Rethinking phase 2 trials in amyotrophic lateral sclerosis.Brain : a journal of neurology · 2025Article
- Revisiting motor unit recruitment to TMS in amyotrophic lateral sclerosis: cortical inhibition is retained during voluntary contractions.Experimental brain research · 2025Article
16 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
23 authors at 1 institution in 6 countries.
Leonard H van den BergFrom the Department of Neurology (L.H.v.d.B.), Brain Center Rudolf Magnus, University Medical Center Utrecht, the Netherlands; Department of Neurology (E.S.), Mayo Clinic, Rochester, MN; Department of Neurology (G.G.), University of Kansas Medical Center, Kansas City; Department of Medicine (E.A.M.), Massachusetts General Hospital, Biostatistics Center, Harvard Medical School, Boston; Department of Neurology (J.A., H.M.), Columbia University, Eleanor and Lou Gehrig ALS Center, New York, NY; Department of Neurology (R.H.B.), Cedars-Sinai Medical Center, Los Angeles, CA; Department of Neurology (M.B.), University of Miami, FL; Neurological Clinical Research Institute (J.D.B.), Massachusetts General Hospital, Boston; Rita Levi Montalcini Department of Neuroscience (A.C.), University of Torino, Italy; Centre Constitutif SLA (P.C.), Université de Tours, France; Department of Neurology (A.G.), Clinical Research Unit, Montreal Neurological Institute, Neurosurgery, McGill University, Montreal, Canada; National Institute of Neurological Disorders and Stroke (A.K.G.), National Institutes of Health, Bethesda, MD; ALS Center (C.L.-H.), University of California San Francisco; Department of Neuroscience (C.J.M.), Sheffield Institute for Translational Neuroscience, University of Sheffield, UK; Department of Neurology (E.P.P.), Section of ALS & Related Disorders, Cleveland Clinic, OH; Department of Neurology (J.R.), The Center for Restorative Neurology, Loma Linda University School of Medicine, CA; Department of Neurology and Laboratory of Neuroscience (V.S.), Istituto Auxologico Italiano, IRCCS, Milan; Department of Pathophysiology and Transplantation (V.S.), "Dino Ferrari" Centre, Università degli Studi di Milano, Milan, Italy; Nuffield Department of Clinical Neurosciences (M.R.T.), University of Oxford, UK; Neuromuscular Diseases Unit/ALS Clinic (M.W.), Kantonsspital St. Gallen, Switzerland; Carolinas Neuromuscular/ALS-MDA Care Center (B.R.B.), Charlotte; Department of Neurology (B.R.B.), Carolinas Medical Center, University of North Carolina School of Medicine, Charlotte; Forbes Norris ALS Treatment and Research Center (R.G.M.), California Pacific Medical Center San Francisco; and Department of Neurosciences (R.G.M.), Stanford University, CA. L.H.vandenBerg@umcutrecht.nl.
Eric SorensonFrom the Department of Neurology (L.H.v.d.B.), Brain Center Rudolf Magnus, University Medical Center Utrecht, the Netherlands; Department of Neurology (E.S.), Mayo Clinic, Rochester, MN; Department of Neurology (G.G.), University of Kansas Medical Center, Kansas City; Department of Medicine (E.A.M.), Massachusetts General Hospital, Biostatistics Center, Harvard Medical School, Boston; Department of Neurology (J.A., H.M.), Columbia University, Eleanor and Lou Gehrig ALS Center, New York, NY; Department of Neurology (R.H.B.), Cedars-Sinai Medical Center, Los Angeles, CA; Department of Neurology (M.B.), University of Miami, FL; Neurological Clinical Research Institute (J.D.B.), Massachusetts General Hospital, Boston; Rita Levi Montalcini Department of Neuroscience (A.C.), University of Torino, Italy; Centre Constitutif SLA (P.C.), Université de Tours, France; Department of Neurology (A.G.), Clinical Research Unit, Montreal Neurological Institute, Neurosurgery, McGill University, Montreal, Canada; National Institute of Neurological Disorders and Stroke (A.K.G.), National Institutes of Health, Bethesda, MD; ALS Center (C.L.-H.), University of California San Francisco; Department of Neuroscience (C.J.M.), Sheffield Institute for Translational Neuroscience, University of Sheffield, UK; Department of Neurology (E.P.P.), Section of ALS & Related Disorders, Cleveland Clinic, OH; Department of Neurology (J.R.), The Center for Restorative Neurology, Loma Linda University School of Medicine, CA; Department of Neurology and Laboratory of Neuroscience (V.S.), Istituto Auxologico Italiano, IRCCS, Milan; Department of Pathophysiology and Transplantation (V.S.), "Dino Ferrari" Centre, Università degli Studi di Milano, Milan, Italy; Nuffield Department of Clinical Neurosciences (M.R.T.), University of Oxford, UK; Neuromuscular Diseases Unit/ALS Clinic (M.W.), Kantonsspital St. Gallen, Switzerland; Carolinas Neuromuscular/ALS-MDA Care Center (B.R.B.), Charlotte; Department of Neurology (B.R.B.), Carolinas Medical Center, University of North Carolina School of Medicine, Charlotte; Forbes Norris ALS Treatment and Research Center (R.G.M.), California Pacific Medical Center San Francisco; and Department of Neurosciences (R.G.M.), Stanford University, CA.
Gary GronsethFrom the Department of Neurology (L.H.v.d.B.), Brain Center Rudolf Magnus, University Medical Center Utrecht, the Netherlands; Department of Neurology (E.S.), Mayo Clinic, Rochester, MN; Department of Neurology (G.G.), University of Kansas Medical Center, Kansas City; Department of Medicine (E.A.M.), Massachusetts General Hospital, Biostatistics Center, Harvard Medical School, Boston; Department of Neurology (J.A., H.M.), Columbia University, Eleanor and Lou Gehrig ALS Center, New York, NY; Department of Neurology (R.H.B.), Cedars-Sinai Medical Center, Los Angeles, CA; Department of Neurology (M.B.), University of Miami, FL; Neurological Clinical Research Institute (J.D.B.), Massachusetts General Hospital, Boston; Rita Levi Montalcini Department of Neuroscience (A.C.), University of Torino, Italy; Centre Constitutif SLA (P.C.), Université de Tours, France; Department of Neurology (A.G.), Clinical Research Unit, Montreal Neurological Institute, Neurosurgery, McGill University, Montreal, Canada; National Institute of Neurological Disorders and Stroke (A.K.G.), National Institutes of Health, Bethesda, MD; ALS Center (C.L.-H.), University of California San Francisco; Department of Neuroscience (C.J.M.), Sheffield Institute for Translational Neuroscience, University of Sheffield, UK; Department of Neurology (E.P.P.), Section of ALS & Related Disorders, Cleveland Clinic, OH; Department of Neurology (J.R.), The Center for Restorative Neurology, Loma Linda University School of Medicine, CA; Department of Neurology and Laboratory of Neuroscience (V.S.), Istituto Auxologico Italiano, IRCCS, Milan; Department of Pathophysiology and Transplantation (V.S.), "Dino Ferrari" Centre, Università degli Studi di Milano, Milan, Italy; Nuffield Department of Clinical Neurosciences (M.R.T.), University of Oxford, UK; Neuromuscular Diseases Unit/ALS Clinic (M.W.), Kantonsspital St. Gallen, Switzerland; Carolinas Neuromuscular/ALS-MDA Care Center (B.R.B.), Charlotte; Department of Neurology (B.R.B.), Carolinas Medical Center, University of North Carolina School of Medicine, Charlotte; Forbes Norris ALS Treatment and Research Center (R.G.M.), California Pacific Medical Center San Francisco; and Department of Neurosciences (R.G.M.), Stanford University, CA.
Eric A MacklinFrom the Department of Neurology (L.H.v.d.B.), Brain Center Rudolf Magnus, University Medical Center Utrecht, the Netherlands; Department of Neurology (E.S.), Mayo Clinic, Rochester, MN; Department of Neurology (G.G.), University of Kansas Medical Center, Kansas City; Department of Medicine (E.A.M.), Massachusetts General Hospital, Biostatistics Center, Harvard Medical School, Boston; Department of Neurology (J.A., H.M.), Columbia University, Eleanor and Lou Gehrig ALS Center, New York, NY; Department of Neurology (R.H.B.), Cedars-Sinai Medical Center, Los Angeles, CA; Department of Neurology (M.B.), University of Miami, FL; Neurological Clinical Research Institute (J.D.B.), Massachusetts General Hospital, Boston; Rita Levi Montalcini Department of Neuroscience (A.C.), University of Torino, Italy; Centre Constitutif SLA (P.C.), Université de Tours, France; Department of Neurology (A.G.), Clinical Research Unit, Montreal Neurological Institute, Neurosurgery, McGill University, Montreal, Canada; National Institute of Neurological Disorders and Stroke (A.K.G.), National Institutes of Health, Bethesda, MD; ALS Center (C.L.-H.), University of California San Francisco; Department of Neuroscience (C.J.M.), Sheffield Institute for Translational Neuroscience, University of Sheffield, UK; Department of Neurology (E.P.P.), Section of ALS & Related Disorders, Cleveland Clinic, OH; Department of Neurology (J.R.), The Center for Restorative Neurology, Loma Linda University School of Medicine, CA; Department of Neurology and Laboratory of Neuroscience (V.S.), Istituto Auxologico Italiano, IRCCS, Milan; Department of Pathophysiology and Transplantation (V.S.), "Dino Ferrari" Centre, Università degli Studi di Milano, Milan, Italy; Nuffield Department of Clinical Neurosciences (M.R.T.), University of Oxford, UK; Neuromuscular Diseases Unit/ALS Clinic (M.W.), Kantonsspital St. Gallen, Switzerland; Carolinas Neuromuscular/ALS-MDA Care Center (B.R.B.), Charlotte; Department of Neurology (B.R.B.), Carolinas Medical Center, University of North Carolina School of Medicine, Charlotte; Forbes Norris ALS Treatment and Research Center (R.G.M.), California Pacific Medical Center San Francisco; and Department of Neurosciences (R.G.M.), Stanford University, CA.
Jinsy AndrewsFrom the Department of Neurology (L.H.v.d.B.), Brain Center Rudolf Magnus, University Medical Center Utrecht, the Netherlands; Department of Neurology (E.S.), Mayo Clinic, Rochester, MN; Department of Neurology (G.G.), University of Kansas Medical Center, Kansas City; Department of Medicine (E.A.M.), Massachusetts General Hospital, Biostatistics Center, Harvard Medical School, Boston; Department of Neurology (J.A., H.M.), Columbia University, Eleanor and Lou Gehrig ALS Center, New York, NY; Department of Neurology (R.H.B.), Cedars-Sinai Medical Center, Los Angeles, CA; Department of Neurology (M.B.), University of Miami, FL; Neurological Clinical Research Institute (J.D.B.), Massachusetts General Hospital, Boston; Rita Levi Montalcini Department of Neuroscience (A.C.), University of Torino, Italy; Centre Constitutif SLA (P.C.), Université de Tours, France; Department of Neurology (A.G.), Clinical Research Unit, Montreal Neurological Institute, Neurosurgery, McGill University, Montreal, Canada; National Institute of Neurological Disorders and Stroke (A.K.G.), National Institutes of Health, Bethesda, MD; ALS Center (C.L.-H.), University of California San Francisco; Department of Neuroscience (C.J.M.), Sheffield Institute for Translational Neuroscience, University of Sheffield, UK; Department of Neurology (E.P.P.), Section of ALS & Related Disorders, Cleveland Clinic, OH; Department of Neurology (J.R.), The Center for Restorative Neurology, Loma Linda University School of Medicine, CA; Department of Neurology and Laboratory of Neuroscience (V.S.), Istituto Auxologico Italiano, IRCCS, Milan; Department of Pathophysiology and Transplantation (V.S.), "Dino Ferrari" Centre, Università degli Studi di Milano, Milan, Italy; Nuffield Department of Clinical Neurosciences (M.R.T.), University of Oxford, UK; Neuromuscular Diseases Unit/ALS Clinic (M.W.), Kantonsspital St. Gallen, Switzerland; Carolinas Neuromuscular/ALS-MDA Care Center (B.R.B.), Charlotte; Department of Neurology (B.R.B.), Carolinas Medical Center, University of North Carolina School of Medicine, Charlotte; Forbes Norris ALS Treatment and Research Center (R.G.M.), California Pacific Medical Center San Francisco; and Department of Neurosciences (R.G.M.), Stanford University, CA.
Robert H BalohFrom the Department of Neurology (L.H.v.d.B.), Brain Center Rudolf Magnus, University Medical Center Utrecht, the Netherlands; Department of Neurology (E.S.), Mayo Clinic, Rochester, MN; Department of Neurology (G.G.), University of Kansas Medical Center, Kansas City; Department of Medicine (E.A.M.), Massachusetts General Hospital, Biostatistics Center, Harvard Medical School, Boston; Department of Neurology (J.A., H.M.), Columbia University, Eleanor and Lou Gehrig ALS Center, New York, NY; Department of Neurology (R.H.B.), Cedars-Sinai Medical Center, Los Angeles, CA; Department of Neurology (M.B.), University of Miami, FL; Neurological Clinical Research Institute (J.D.B.), Massachusetts General Hospital, Boston; Rita Levi Montalcini Department of Neuroscience (A.C.), University of Torino, Italy; Centre Constitutif SLA (P.C.), Université de Tours, France; Department of Neurology (A.G.), Clinical Research Unit, Montreal Neurological Institute, Neurosurgery, McGill University, Montreal, Canada; National Institute of Neurological Disorders and Stroke (A.K.G.), National Institutes of Health, Bethesda, MD; ALS Center (C.L.-H.), University of California San Francisco; Department of Neuroscience (C.J.M.), Sheffield Institute for Translational Neuroscience, University of Sheffield, UK; Department of Neurology (E.P.P.), Section of ALS & Related Disorders, Cleveland Clinic, OH; Department of Neurology (J.R.), The Center for Restorative Neurology, Loma Linda University School of Medicine, CA; Department of Neurology and Laboratory of Neuroscience (V.S.), Istituto Auxologico Italiano, IRCCS, Milan; Department of Pathophysiology and Transplantation (V.S.), "Dino Ferrari" Centre, Università degli Studi di Milano, Milan, Italy; Nuffield Department of Clinical Neurosciences (M.R.T.), University of Oxford, UK; Neuromuscular Diseases Unit/ALS Clinic (M.W.), Kantonsspital St. Gallen, Switzerland; Carolinas Neuromuscular/ALS-MDA Care Center (B.R.B.), Charlotte; Department of Neurology (B.R.B.), Carolinas Medical Center, University of North Carolina School of Medicine, Charlotte; Forbes Norris ALS Treatment and Research Center (R.G.M.), California Pacific Medical Center San Francisco; and Department of Neurosciences (R.G.M.), Stanford University, CA.
Michael BenatarFrom the Department of Neurology (L.H.v.d.B.), Brain Center Rudolf Magnus, University Medical Center Utrecht, the Netherlands; Department of Neurology (E.S.), Mayo Clinic, Rochester, MN; Department of Neurology (G.G.), University of Kansas Medical Center, Kansas City; Department of Medicine (E.A.M.), Massachusetts General Hospital, Biostatistics Center, Harvard Medical School, Boston; Department of Neurology (J.A., H.M.), Columbia University, Eleanor and Lou Gehrig ALS Center, New York, NY; Department of Neurology (R.H.B.), Cedars-Sinai Medical Center, Los Angeles, CA; Department of Neurology (M.B.), University of Miami, FL; Neurological Clinical Research Institute (J.D.B.), Massachusetts General Hospital, Boston; Rita Levi Montalcini Department of Neuroscience (A.C.), University of Torino, Italy; Centre Constitutif SLA (P.C.), Université de Tours, France; Department of Neurology (A.G.), Clinical Research Unit, Montreal Neurological Institute, Neurosurgery, McGill University, Montreal, Canada; National Institute of Neurological Disorders and Stroke (A.K.G.), National Institutes of Health, Bethesda, MD; ALS Center (C.L.-H.), University of California San Francisco; Department of Neuroscience (C.J.M.), Sheffield Institute for Translational Neuroscience, University of Sheffield, UK; Department of Neurology (E.P.P.), Section of ALS & Related Disorders, Cleveland Clinic, OH; Department of Neurology (J.R.), The Center for Restorative Neurology, Loma Linda University School of Medicine, CA; Department of Neurology and Laboratory of Neuroscience (V.S.), Istituto Auxologico Italiano, IRCCS, Milan; Department of Pathophysiology and Transplantation (V.S.), "Dino Ferrari" Centre, Università degli Studi di Milano, Milan, Italy; Nuffield Department of Clinical Neurosciences (M.R.T.), University of Oxford, UK; Neuromuscular Diseases Unit/ALS Clinic (M.W.), Kantonsspital St. Gallen, Switzerland; Carolinas Neuromuscular/ALS-MDA Care Center (B.R.B.), Charlotte; Department of Neurology (B.R.B.), Carolinas Medical Center, University of North Carolina School of Medicine, Charlotte; Forbes Norris ALS Treatment and Research Center (R.G.M.), California Pacific Medical Center San Francisco; and Department of Neurosciences (R.G.M.), Stanford University, CA.
James D BerryFrom the Department of Neurology (L.H.v.d.B.), Brain Center Rudolf Magnus, University Medical Center Utrecht, the Netherlands; Department of Neurology (E.S.), Mayo Clinic, Rochester, MN; Department of Neurology (G.G.), University of Kansas Medical Center, Kansas City; Department of Medicine (E.A.M.), Massachusetts General Hospital, Biostatistics Center, Harvard Medical School, Boston; Department of Neurology (J.A., H.M.), Columbia University, Eleanor and Lou Gehrig ALS Center, New York, NY; Department of Neurology (R.H.B.), Cedars-Sinai Medical Center, Los Angeles, CA; Department of Neurology (M.B.), University of Miami, FL; Neurological Clinical Research Institute (J.D.B.), Massachusetts General Hospital, Boston; Rita Levi Montalcini Department of Neuroscience (A.C.), University of Torino, Italy; Centre Constitutif SLA (P.C.), Université de Tours, France; Department of Neurology (A.G.), Clinical Research Unit, Montreal Neurological Institute, Neurosurgery, McGill University, Montreal, Canada; National Institute of Neurological Disorders and Stroke (A.K.G.), National Institutes of Health, Bethesda, MD; ALS Center (C.L.-H.), University of California San Francisco; Department of Neuroscience (C.J.M.), Sheffield Institute for Translational Neuroscience, University of Sheffield, UK; Department of Neurology (E.P.P.), Section of ALS & Related Disorders, Cleveland Clinic, OH; Department of Neurology (J.R.), The Center for Restorative Neurology, Loma Linda University School of Medicine, CA; Department of Neurology and Laboratory of Neuroscience (V.S.), Istituto Auxologico Italiano, IRCCS, Milan; Department of Pathophysiology and Transplantation (V.S.), "Dino Ferrari" Centre, Università degli Studi di Milano, Milan, Italy; Nuffield Department of Clinical Neurosciences (M.R.T.), University of Oxford, UK; Neuromuscular Diseases Unit/ALS Clinic (M.W.), Kantonsspital St. Gallen, Switzerland; Carolinas Neuromuscular/ALS-MDA Care Center (B.R.B.), Charlotte; Department of Neurology (B.R.B.), Carolinas Medical Center, University of North Carolina School of Medicine, Charlotte; Forbes Norris ALS Treatment and Research Center (R.G.M.), California Pacific Medical Center San Francisco; and Department of Neurosciences (R.G.M.), Stanford University, CA.
Adriano ChioFrom the Department of Neurology (L.H.v.d.B.), Brain Center Rudolf Magnus, University Medical Center Utrecht, the Netherlands; Department of Neurology (E.S.), Mayo Clinic, Rochester, MN; Department of Neurology (G.G.), University of Kansas Medical Center, Kansas City; Department of Medicine (E.A.M.), Massachusetts General Hospital, Biostatistics Center, Harvard Medical School, Boston; Department of Neurology (J.A., H.M.), Columbia University, Eleanor and Lou Gehrig ALS Center, New York, NY; Department of Neurology (R.H.B.), Cedars-Sinai Medical Center, Los Angeles, CA; Department of Neurology (M.B.), University of Miami, FL; Neurological Clinical Research Institute (J.D.B.), Massachusetts General Hospital, Boston; Rita Levi Montalcini Department of Neuroscience (A.C.), University of Torino, Italy; Centre Constitutif SLA (P.C.), Université de Tours, France; Department of Neurology (A.G.), Clinical Research Unit, Montreal Neurological Institute, Neurosurgery, McGill University, Montreal, Canada; National Institute of Neurological Disorders and Stroke (A.K.G.), National Institutes of Health, Bethesda, MD; ALS Center (C.L.-H.), University of California San Francisco; Department of Neuroscience (C.J.M.), Sheffield Institute for Translational Neuroscience, University of Sheffield, UK; Department of Neurology (E.P.P.), Section of ALS & Related Disorders, Cleveland Clinic, OH; Department of Neurology (J.R.), The Center for Restorative Neurology, Loma Linda University School of Medicine, CA; Department of Neurology and Laboratory of Neuroscience (V.S.), Istituto Auxologico Italiano, IRCCS, Milan; Department of Pathophysiology and Transplantation (V.S.), "Dino Ferrari" Centre, Università degli Studi di Milano, Milan, Italy; Nuffield Department of Clinical Neurosciences (M.R.T.), University of Oxford, UK; Neuromuscular Diseases Unit/ALS Clinic (M.W.), Kantonsspital St. Gallen, Switzerland; Carolinas Neuromuscular/ALS-MDA Care Center (B.R.B.), Charlotte; Department of Neurology (B.R.B.), Carolinas Medical Center, University of North Carolina School of Medicine, Charlotte; Forbes Norris ALS Treatment and Research Center (R.G.M.), California Pacific Medical Center San Francisco; and Department of Neurosciences (R.G.M.), Stanford University, CA.ORCID 0000-0001-9579-5341
Philippe CorciaFrom the Department of Neurology (L.H.v.d.B.), Brain Center Rudolf Magnus, University Medical Center Utrecht, the Netherlands; Department of Neurology (E.S.), Mayo Clinic, Rochester, MN; Department of Neurology (G.G.), University of Kansas Medical Center, Kansas City; Department of Medicine (E.A.M.), Massachusetts General Hospital, Biostatistics Center, Harvard Medical School, Boston; Department of Neurology (J.A., H.M.), Columbia University, Eleanor and Lou Gehrig ALS Center, New York, NY; Department of Neurology (R.H.B.), Cedars-Sinai Medical Center, Los Angeles, CA; Department of Neurology (M.B.), University of Miami, FL; Neurological Clinical Research Institute (J.D.B.), Massachusetts General Hospital, Boston; Rita Levi Montalcini Department of Neuroscience (A.C.), University of Torino, Italy; Centre Constitutif SLA (P.C.), Université de Tours, France; Department of Neurology (A.G.), Clinical Research Unit, Montreal Neurological Institute, Neurosurgery, McGill University, Montreal, Canada; National Institute of Neurological Disorders and Stroke (A.K.G.), National Institutes of Health, Bethesda, MD; ALS Center (C.L.-H.), University of California San Francisco; Department of Neuroscience (C.J.M.), Sheffield Institute for Translational Neuroscience, University of Sheffield, UK; Department of Neurology (E.P.P.), Section of ALS & Related Disorders, Cleveland Clinic, OH; Department of Neurology (J.R.), The Center for Restorative Neurology, Loma Linda University School of Medicine, CA; Department of Neurology and Laboratory of Neuroscience (V.S.), Istituto Auxologico Italiano, IRCCS, Milan; Department of Pathophysiology and Transplantation (V.S.), "Dino Ferrari" Centre, Università degli Studi di Milano, Milan, Italy; Nuffield Department of Clinical Neurosciences (M.R.T.), University of Oxford, UK; Neuromuscular Diseases Unit/ALS Clinic (M.W.), Kantonsspital St. Gallen, Switzerland; Carolinas Neuromuscular/ALS-MDA Care Center (B.R.B.), Charlotte; Department of Neurology (B.R.B.), Carolinas Medical Center, University of North Carolina School of Medicine, Charlotte; Forbes Norris ALS Treatment and Research Center (R.G.M.), California Pacific Medical Center San Francisco; and Department of Neurosciences (R.G.M.), Stanford University, CA.
Angela GengeFrom the Department of Neurology (L.H.v.d.B.), Brain Center Rudolf Magnus, University Medical Center Utrecht, the Netherlands; Department of Neurology (E.S.), Mayo Clinic, Rochester, MN; Department of Neurology (G.G.), University of Kansas Medical Center, Kansas City; Department of Medicine (E.A.M.), Massachusetts General Hospital, Biostatistics Center, Harvard Medical School, Boston; Department of Neurology (J.A., H.M.), Columbia University, Eleanor and Lou Gehrig ALS Center, New York, NY; Department of Neurology (R.H.B.), Cedars-Sinai Medical Center, Los Angeles, CA; Department of Neurology (M.B.), University of Miami, FL; Neurological Clinical Research Institute (J.D.B.), Massachusetts General Hospital, Boston; Rita Levi Montalcini Department of Neuroscience (A.C.), University of Torino, Italy; Centre Constitutif SLA (P.C.), Université de Tours, France; Department of Neurology (A.G.), Clinical Research Unit, Montreal Neurological Institute, Neurosurgery, McGill University, Montreal, Canada; National Institute of Neurological Disorders and Stroke (A.K.G.), National Institutes of Health, Bethesda, MD; ALS Center (C.L.-H.), University of California San Francisco; Department of Neuroscience (C.J.M.), Sheffield Institute for Translational Neuroscience, University of Sheffield, UK; Department of Neurology (E.P.P.), Section of ALS & Related Disorders, Cleveland Clinic, OH; Department of Neurology (J.R.), The Center for Restorative Neurology, Loma Linda University School of Medicine, CA; Department of Neurology and Laboratory of Neuroscience (V.S.), Istituto Auxologico Italiano, IRCCS, Milan; Department of Pathophysiology and Transplantation (V.S.), "Dino Ferrari" Centre, Università degli Studi di Milano, Milan, Italy; Nuffield Department of Clinical Neurosciences (M.R.T.), University of Oxford, UK; Neuromuscular Diseases Unit/ALS Clinic (M.W.), Kantonsspital St. Gallen, Switzerland; Carolinas Neuromuscular/ALS-MDA Care Center (B.R.B.), Charlotte; Department of Neurology (B.R.B.), Carolinas Medical Center, University of North Carolina School of Medicine, Charlotte; Forbes Norris ALS Treatment and Research Center (R.G.M.), California Pacific Medical Center San Francisco; and Department of Neurosciences (R.G.M.), Stanford University, CA.
Amelie K GubitzFrom the Department of Neurology (L.H.v.d.B.), Brain Center Rudolf Magnus, University Medical Center Utrecht, the Netherlands; Department of Neurology (E.S.), Mayo Clinic, Rochester, MN; Department of Neurology (G.G.), University of Kansas Medical Center, Kansas City; Department of Medicine (E.A.M.), Massachusetts General Hospital, Biostatistics Center, Harvard Medical School, Boston; Department of Neurology (J.A., H.M.), Columbia University, Eleanor and Lou Gehrig ALS Center, New York, NY; Department of Neurology (R.H.B.), Cedars-Sinai Medical Center, Los Angeles, CA; Department of Neurology (M.B.), University of Miami, FL; Neurological Clinical Research Institute (J.D.B.), Massachusetts General Hospital, Boston; Rita Levi Montalcini Department of Neuroscience (A.C.), University of Torino, Italy; Centre Constitutif SLA (P.C.), Université de Tours, France; Department of Neurology (A.G.), Clinical Research Unit, Montreal Neurological Institute, Neurosurgery, McGill University, Montreal, Canada; National Institute of Neurological Disorders and Stroke (A.K.G.), National Institutes of Health, Bethesda, MD; ALS Center (C.L.-H.), University of California San Francisco; Department of Neuroscience (C.J.M.), Sheffield Institute for Translational Neuroscience, University of Sheffield, UK; Department of Neurology (E.P.P.), Section of ALS & Related Disorders, Cleveland Clinic, OH; Department of Neurology (J.R.), The Center for Restorative Neurology, Loma Linda University School of Medicine, CA; Department of Neurology and Laboratory of Neuroscience (V.S.), Istituto Auxologico Italiano, IRCCS, Milan; Department of Pathophysiology and Transplantation (V.S.), "Dino Ferrari" Centre, Università degli Studi di Milano, Milan, Italy; Nuffield Department of Clinical Neurosciences (M.R.T.), University of Oxford, UK; Neuromuscular Diseases Unit/ALS Clinic (M.W.), Kantonsspital St. Gallen, Switzerland; Carolinas Neuromuscular/ALS-MDA Care Center (B.R.B.), Charlotte; Department of Neurology (B.R.B.), Carolinas Medical Center, University of North Carolina School of Medicine, Charlotte; Forbes Norris ALS Treatment and Research Center (R.G.M.), California Pacific Medical Center San Francisco; and Department of Neurosciences (R.G.M.), Stanford University, CA.
Catherine Lomen-HoerthFrom the Department of Neurology (L.H.v.d.B.), Brain Center Rudolf Magnus, University Medical Center Utrecht, the Netherlands; Department of Neurology (E.S.), Mayo Clinic, Rochester, MN; Department of Neurology (G.G.), University of Kansas Medical Center, Kansas City; Department of Medicine (E.A.M.), Massachusetts General Hospital, Biostatistics Center, Harvard Medical School, Boston; Department of Neurology (J.A., H.M.), Columbia University, Eleanor and Lou Gehrig ALS Center, New York, NY; Department of Neurology (R.H.B.), Cedars-Sinai Medical Center, Los Angeles, CA; Department of Neurology (M.B.), University of Miami, FL; Neurological Clinical Research Institute (J.D.B.), Massachusetts General Hospital, Boston; Rita Levi Montalcini Department of Neuroscience (A.C.), University of Torino, Italy; Centre Constitutif SLA (P.C.), Université de Tours, France; Department of Neurology (A.G.), Clinical Research Unit, Montreal Neurological Institute, Neurosurgery, McGill University, Montreal, Canada; National Institute of Neurological Disorders and Stroke (A.K.G.), National Institutes of Health, Bethesda, MD; ALS Center (C.L.-H.), University of California San Francisco; Department of Neuroscience (C.J.M.), Sheffield Institute for Translational Neuroscience, University of Sheffield, UK; Department of Neurology (E.P.P.), Section of ALS & Related Disorders, Cleveland Clinic, OH; Department of Neurology (J.R.), The Center for Restorative Neurology, Loma Linda University School of Medicine, CA; Department of Neurology and Laboratory of Neuroscience (V.S.), Istituto Auxologico Italiano, IRCCS, Milan; Department of Pathophysiology and Transplantation (V.S.), "Dino Ferrari" Centre, Università degli Studi di Milano, Milan, Italy; Nuffield Department of Clinical Neurosciences (M.R.T.), University of Oxford, UK; Neuromuscular Diseases Unit/ALS Clinic (M.W.), Kantonsspital St. Gallen, Switzerland; Carolinas Neuromuscular/ALS-MDA Care Center (B.R.B.), Charlotte; Department of Neurology (B.R.B.), Carolinas Medical Center, University of North Carolina School of Medicine, Charlotte; Forbes Norris ALS Treatment and Research Center (R.G.M.), California Pacific Medical Center San Francisco; and Department of Neurosciences (R.G.M.), Stanford University, CA.
Christopher J McDermottFrom the Department of Neurology (L.H.v.d.B.), Brain Center Rudolf Magnus, University Medical Center Utrecht, the Netherlands; Department of Neurology (E.S.), Mayo Clinic, Rochester, MN; Department of Neurology (G.G.), University of Kansas Medical Center, Kansas City; Department of Medicine (E.A.M.), Massachusetts General Hospital, Biostatistics Center, Harvard Medical School, Boston; Department of Neurology (J.A., H.M.), Columbia University, Eleanor and Lou Gehrig ALS Center, New York, NY; Department of Neurology (R.H.B.), Cedars-Sinai Medical Center, Los Angeles, CA; Department of Neurology (M.B.), University of Miami, FL; Neurological Clinical Research Institute (J.D.B.), Massachusetts General Hospital, Boston; Rita Levi Montalcini Department of Neuroscience (A.C.), University of Torino, Italy; Centre Constitutif SLA (P.C.), Université de Tours, France; Department of Neurology (A.G.), Clinical Research Unit, Montreal Neurological Institute, Neurosurgery, McGill University, Montreal, Canada; National Institute of Neurological Disorders and Stroke (A.K.G.), National Institutes of Health, Bethesda, MD; ALS Center (C.L.-H.), University of California San Francisco; Department of Neuroscience (C.J.M.), Sheffield Institute for Translational Neuroscience, University of Sheffield, UK; Department of Neurology (E.P.P.), Section of ALS & Related Disorders, Cleveland Clinic, OH; Department of Neurology (J.R.), The Center for Restorative Neurology, Loma Linda University School of Medicine, CA; Department of Neurology and Laboratory of Neuroscience (V.S.), Istituto Auxologico Italiano, IRCCS, Milan; Department of Pathophysiology and Transplantation (V.S.), "Dino Ferrari" Centre, Università degli Studi di Milano, Milan, Italy; Nuffield Department of Clinical Neurosciences (M.R.T.), University of Oxford, UK; Neuromuscular Diseases Unit/ALS Clinic (M.W.), Kantonsspital St. Gallen, Switzerland; Carolinas Neuromuscular/ALS-MDA Care Center (B.R.B.), Charlotte; Department of Neurology (B.R.B.), Carolinas Medical Center, University of North Carolina School of Medicine, Charlotte; Forbes Norris ALS Treatment and Research Center (R.G.M.), California Pacific Medical Center San Francisco; and Department of Neurosciences (R.G.M.), Stanford University, CA.
Erik P PioroFrom the Department of Neurology (L.H.v.d.B.), Brain Center Rudolf Magnus, University Medical Center Utrecht, the Netherlands; Department of Neurology (E.S.), Mayo Clinic, Rochester, MN; Department of Neurology (G.G.), University of Kansas Medical Center, Kansas City; Department of Medicine (E.A.M.), Massachusetts General Hospital, Biostatistics Center, Harvard Medical School, Boston; Department of Neurology (J.A., H.M.), Columbia University, Eleanor and Lou Gehrig ALS Center, New York, NY; Department of Neurology (R.H.B.), Cedars-Sinai Medical Center, Los Angeles, CA; Department of Neurology (M.B.), University of Miami, FL; Neurological Clinical Research Institute (J.D.B.), Massachusetts General Hospital, Boston; Rita Levi Montalcini Department of Neuroscience (A.C.), University of Torino, Italy; Centre Constitutif SLA (P.C.), Université de Tours, France; Department of Neurology (A.G.), Clinical Research Unit, Montreal Neurological Institute, Neurosurgery, McGill University, Montreal, Canada; National Institute of Neurological Disorders and Stroke (A.K.G.), National Institutes of Health, Bethesda, MD; ALS Center (C.L.-H.), University of California San Francisco; Department of Neuroscience (C.J.M.), Sheffield Institute for Translational Neuroscience, University of Sheffield, UK; Department of Neurology (E.P.P.), Section of ALS & Related Disorders, Cleveland Clinic, OH; Department of Neurology (J.R.), The Center for Restorative Neurology, Loma Linda University School of Medicine, CA; Department of Neurology and Laboratory of Neuroscience (V.S.), Istituto Auxologico Italiano, IRCCS, Milan; Department of Pathophysiology and Transplantation (V.S.), "Dino Ferrari" Centre, Università degli Studi di Milano, Milan, Italy; Nuffield Department of Clinical Neurosciences (M.R.T.), University of Oxford, UK; Neuromuscular Diseases Unit/ALS Clinic (M.W.), Kantonsspital St. Gallen, Switzerland; Carolinas Neuromuscular/ALS-MDA Care Center (B.R.B.), Charlotte; Department of Neurology (B.R.B.), Carolinas Medical Center, University of North Carolina School of Medicine, Charlotte; Forbes Norris ALS Treatment and Research Center (R.G.M.), California Pacific Medical Center San Francisco; and Department of Neurosciences (R.G.M.), Stanford University, CA.
Jeffrey RosenfeldFrom the Department of Neurology (L.H.v.d.B.), Brain Center Rudolf Magnus, University Medical Center Utrecht, the Netherlands; Department of Neurology (E.S.), Mayo Clinic, Rochester, MN; Department of Neurology (G.G.), University of Kansas Medical Center, Kansas City; Department of Medicine (E.A.M.), Massachusetts General Hospital, Biostatistics Center, Harvard Medical School, Boston; Department of Neurology (J.A., H.M.), Columbia University, Eleanor and Lou Gehrig ALS Center, New York, NY; Department of Neurology (R.H.B.), Cedars-Sinai Medical Center, Los Angeles, CA; Department of Neurology (M.B.), University of Miami, FL; Neurological Clinical Research Institute (J.D.B.), Massachusetts General Hospital, Boston; Rita Levi Montalcini Department of Neuroscience (A.C.), University of Torino, Italy; Centre Constitutif SLA (P.C.), Université de Tours, France; Department of Neurology (A.G.), Clinical Research Unit, Montreal Neurological Institute, Neurosurgery, McGill University, Montreal, Canada; National Institute of Neurological Disorders and Stroke (A.K.G.), National Institutes of Health, Bethesda, MD; ALS Center (C.L.-H.), University of California San Francisco; Department of Neuroscience (C.J.M.), Sheffield Institute for Translational Neuroscience, University of Sheffield, UK; Department of Neurology (E.P.P.), Section of ALS & Related Disorders, Cleveland Clinic, OH; Department of Neurology (J.R.), The Center for Restorative Neurology, Loma Linda University School of Medicine, CA; Department of Neurology and Laboratory of Neuroscience (V.S.), Istituto Auxologico Italiano, IRCCS, Milan; Department of Pathophysiology and Transplantation (V.S.), "Dino Ferrari" Centre, Università degli Studi di Milano, Milan, Italy; Nuffield Department of Clinical Neurosciences (M.R.T.), University of Oxford, UK; Neuromuscular Diseases Unit/ALS Clinic (M.W.), Kantonsspital St. Gallen, Switzerland; Carolinas Neuromuscular/ALS-MDA Care Center (B.R.B.), Charlotte; Department of Neurology (B.R.B.), Carolinas Medical Center, University of North Carolina School of Medicine, Charlotte; Forbes Norris ALS Treatment and Research Center (R.G.M.), California Pacific Medical Center San Francisco; and Department of Neurosciences (R.G.M.), Stanford University, CA.ORCID 0000-0003-4327-7774
Vincenzo SilaniFrom the Department of Neurology (L.H.v.d.B.), Brain Center Rudolf Magnus, University Medical Center Utrecht, the Netherlands; Department of Neurology (E.S.), Mayo Clinic, Rochester, MN; Department of Neurology (G.G.), University of Kansas Medical Center, Kansas City; Department of Medicine (E.A.M.), Massachusetts General Hospital, Biostatistics Center, Harvard Medical School, Boston; Department of Neurology (J.A., H.M.), Columbia University, Eleanor and Lou Gehrig ALS Center, New York, NY; Department of Neurology (R.H.B.), Cedars-Sinai Medical Center, Los Angeles, CA; Department of Neurology (M.B.), University of Miami, FL; Neurological Clinical Research Institute (J.D.B.), Massachusetts General Hospital, Boston; Rita Levi Montalcini Department of Neuroscience (A.C.), University of Torino, Italy; Centre Constitutif SLA (P.C.), Université de Tours, France; Department of Neurology (A.G.), Clinical Research Unit, Montreal Neurological Institute, Neurosurgery, McGill University, Montreal, Canada; National Institute of Neurological Disorders and Stroke (A.K.G.), National Institutes of Health, Bethesda, MD; ALS Center (C.L.-H.), University of California San Francisco; Department of Neuroscience (C.J.M.), Sheffield Institute for Translational Neuroscience, University of Sheffield, UK; Department of Neurology (E.P.P.), Section of ALS & Related Disorders, Cleveland Clinic, OH; Department of Neurology (J.R.), The Center for Restorative Neurology, Loma Linda University School of Medicine, CA; Department of Neurology and Laboratory of Neuroscience (V.S.), Istituto Auxologico Italiano, IRCCS, Milan; Department of Pathophysiology and Transplantation (V.S.), "Dino Ferrari" Centre, Università degli Studi di Milano, Milan, Italy; Nuffield Department of Clinical Neurosciences (M.R.T.), University of Oxford, UK; Neuromuscular Diseases Unit/ALS Clinic (M.W.), Kantonsspital St. Gallen, Switzerland; Carolinas Neuromuscular/ALS-MDA Care Center (B.R.B.), Charlotte; Department of Neurology (B.R.B.), Carolinas Medical Center, University of North Carolina School of Medicine, Charlotte; Forbes Norris ALS Treatment and Research Center (R.G.M.), California Pacific Medical Center San Francisco; and Department of Neurosciences (R.G.M.), Stanford University, CA.
Martin R TurnerFrom the Department of Neurology (L.H.v.d.B.), Brain Center Rudolf Magnus, University Medical Center Utrecht, the Netherlands; Department of Neurology (E.S.), Mayo Clinic, Rochester, MN; Department of Neurology (G.G.), University of Kansas Medical Center, Kansas City; Department of Medicine (E.A.M.), Massachusetts General Hospital, Biostatistics Center, Harvard Medical School, Boston; Department of Neurology (J.A., H.M.), Columbia University, Eleanor and Lou Gehrig ALS Center, New York, NY; Department of Neurology (R.H.B.), Cedars-Sinai Medical Center, Los Angeles, CA; Department of Neurology (M.B.), University of Miami, FL; Neurological Clinical Research Institute (J.D.B.), Massachusetts General Hospital, Boston; Rita Levi Montalcini Department of Neuroscience (A.C.), University of Torino, Italy; Centre Constitutif SLA (P.C.), Université de Tours, France; Department of Neurology (A.G.), Clinical Research Unit, Montreal Neurological Institute, Neurosurgery, McGill University, Montreal, Canada; National Institute of Neurological Disorders and Stroke (A.K.G.), National Institutes of Health, Bethesda, MD; ALS Center (C.L.-H.), University of California San Francisco; Department of Neuroscience (C.J.M.), Sheffield Institute for Translational Neuroscience, University of Sheffield, UK; Department of Neurology (E.P.P.), Section of ALS & Related Disorders, Cleveland Clinic, OH; Department of Neurology (J.R.), The Center for Restorative Neurology, Loma Linda University School of Medicine, CA; Department of Neurology and Laboratory of Neuroscience (V.S.), Istituto Auxologico Italiano, IRCCS, Milan; Department of Pathophysiology and Transplantation (V.S.), "Dino Ferrari" Centre, Università degli Studi di Milano, Milan, Italy; Nuffield Department of Clinical Neurosciences (M.R.T.), University of Oxford, UK; Neuromuscular Diseases Unit/ALS Clinic (M.W.), Kantonsspital St. Gallen, Switzerland; Carolinas Neuromuscular/ALS-MDA Care Center (B.R.B.), Charlotte; Department of Neurology (B.R.B.), Carolinas Medical Center, University of North Carolina School of Medicine, Charlotte; Forbes Norris ALS Treatment and Research Center (R.G.M.), California Pacific Medical Center San Francisco; and Department of Neurosciences (R.G.M.), Stanford University, CA.
Markus WeberFrom the Department of Neurology (L.H.v.d.B.), Brain Center Rudolf Magnus, University Medical Center Utrecht, the Netherlands; Department of Neurology (E.S.), Mayo Clinic, Rochester, MN; Department of Neurology (G.G.), University of Kansas Medical Center, Kansas City; Department of Medicine (E.A.M.), Massachusetts General Hospital, Biostatistics Center, Harvard Medical School, Boston; Department of Neurology (J.A., H.M.), Columbia University, Eleanor and Lou Gehrig ALS Center, New York, NY; Department of Neurology (R.H.B.), Cedars-Sinai Medical Center, Los Angeles, CA; Department of Neurology (M.B.), University of Miami, FL; Neurological Clinical Research Institute (J.D.B.), Massachusetts General Hospital, Boston; Rita Levi Montalcini Department of Neuroscience (A.C.), University of Torino, Italy; Centre Constitutif SLA (P.C.), Université de Tours, France; Department of Neurology (A.G.), Clinical Research Unit, Montreal Neurological Institute, Neurosurgery, McGill University, Montreal, Canada; National Institute of Neurological Disorders and Stroke (A.K.G.), National Institutes of Health, Bethesda, MD; ALS Center (C.L.-H.), University of California San Francisco; Department of Neuroscience (C.J.M.), Sheffield Institute for Translational Neuroscience, University of Sheffield, UK; Department of Neurology (E.P.P.), Section of ALS & Related Disorders, Cleveland Clinic, OH; Department of Neurology (J.R.), The Center for Restorative Neurology, Loma Linda University School of Medicine, CA; Department of Neurology and Laboratory of Neuroscience (V.S.), Istituto Auxologico Italiano, IRCCS, Milan; Department of Pathophysiology and Transplantation (V.S.), "Dino Ferrari" Centre, Università degli Studi di Milano, Milan, Italy; Nuffield Department of Clinical Neurosciences (M.R.T.), University of Oxford, UK; Neuromuscular Diseases Unit/ALS Clinic (M.W.), Kantonsspital St. Gallen, Switzerland; Carolinas Neuromuscular/ALS-MDA Care Center (B.R.B.), Charlotte; Department of Neurology (B.R.B.), Carolinas Medical Center, University of North Carolina School of Medicine, Charlotte; Forbes Norris ALS Treatment and Research Center (R.G.M.), California Pacific Medical Center San Francisco; and Department of Neurosciences (R.G.M.), Stanford University, CA.
Benjamin Rix BrooksFrom the Department of Neurology (L.H.v.d.B.), Brain Center Rudolf Magnus, University Medical Center Utrecht, the Netherlands; Department of Neurology (E.S.), Mayo Clinic, Rochester, MN; Department of Neurology (G.G.), University of Kansas Medical Center, Kansas City; Department of Medicine (E.A.M.), Massachusetts General Hospital, Biostatistics Center, Harvard Medical School, Boston; Department of Neurology (J.A., H.M.), Columbia University, Eleanor and Lou Gehrig ALS Center, New York, NY; Department of Neurology (R.H.B.), Cedars-Sinai Medical Center, Los Angeles, CA; Department of Neurology (M.B.), University of Miami, FL; Neurological Clinical Research Institute (J.D.B.), Massachusetts General Hospital, Boston; Rita Levi Montalcini Department of Neuroscience (A.C.), University of Torino, Italy; Centre Constitutif SLA (P.C.), Université de Tours, France; Department of Neurology (A.G.), Clinical Research Unit, Montreal Neurological Institute, Neurosurgery, McGill University, Montreal, Canada; National Institute of Neurological Disorders and Stroke (A.K.G.), National Institutes of Health, Bethesda, MD; ALS Center (C.L.-H.), University of California San Francisco; Department of Neuroscience (C.J.M.), Sheffield Institute for Translational Neuroscience, University of Sheffield, UK; Department of Neurology (E.P.P.), Section of ALS & Related Disorders, Cleveland Clinic, OH; Department of Neurology (J.R.), The Center for Restorative Neurology, Loma Linda University School of Medicine, CA; Department of Neurology and Laboratory of Neuroscience (V.S.), Istituto Auxologico Italiano, IRCCS, Milan; Department of Pathophysiology and Transplantation (V.S.), "Dino Ferrari" Centre, Università degli Studi di Milano, Milan, Italy; Nuffield Department of Clinical Neurosciences (M.R.T.), University of Oxford, UK; Neuromuscular Diseases Unit/ALS Clinic (M.W.), Kantonsspital St. Gallen, Switzerland; Carolinas Neuromuscular/ALS-MDA Care Center (B.R.B.), Charlotte; Department of Neurology (B.R.B.), Carolinas Medical Center, University of North Carolina School of Medicine, Charlotte; Forbes Norris ALS Treatment and Research Center (R.G.M.), California Pacific Medical Center San Francisco; and Department of Neurosciences (R.G.M.), Stanford University, CA.
Robert G MillerFrom the Department of Neurology (L.H.v.d.B.), Brain Center Rudolf Magnus, University Medical Center Utrecht, the Netherlands; Department of Neurology (E.S.), Mayo Clinic, Rochester, MN; Department of Neurology (G.G.), University of Kansas Medical Center, Kansas City; Department of Medicine (E.A.M.), Massachusetts General Hospital, Biostatistics Center, Harvard Medical School, Boston; Department of Neurology (J.A., H.M.), Columbia University, Eleanor and Lou Gehrig ALS Center, New York, NY; Department of Neurology (R.H.B.), Cedars-Sinai Medical Center, Los Angeles, CA; Department of Neurology (M.B.), University of Miami, FL; Neurological Clinical Research Institute (J.D.B.), Massachusetts General Hospital, Boston; Rita Levi Montalcini Department of Neuroscience (A.C.), University of Torino, Italy; Centre Constitutif SLA (P.C.), Université de Tours, France; Department of Neurology (A.G.), Clinical Research Unit, Montreal Neurological Institute, Neurosurgery, McGill University, Montreal, Canada; National Institute of Neurological Disorders and Stroke (A.K.G.), National Institutes of Health, Bethesda, MD; ALS Center (C.L.-H.), University of California San Francisco; Department of Neuroscience (C.J.M.), Sheffield Institute for Translational Neuroscience, University of Sheffield, UK; Department of Neurology (E.P.P.), Section of ALS & Related Disorders, Cleveland Clinic, OH; Department of Neurology (J.R.), The Center for Restorative Neurology, Loma Linda University School of Medicine, CA; Department of Neurology and Laboratory of Neuroscience (V.S.), Istituto Auxologico Italiano, IRCCS, Milan; Department of Pathophysiology and Transplantation (V.S.), "Dino Ferrari" Centre, Università degli Studi di Milano, Milan, Italy; Nuffield Department of Clinical Neurosciences (M.R.T.), University of Oxford, UK; Neuromuscular Diseases Unit/ALS Clinic (M.W.), Kantonsspital St. Gallen, Switzerland; Carolinas Neuromuscular/ALS-MDA Care Center (B.R.B.), Charlotte; Department of Neurology (B.R.B.), Carolinas Medical Center, University of North Carolina School of Medicine, Charlotte; Forbes Norris ALS Treatment and Research Center (R.G.M.), California Pacific Medical Center San Francisco; and Department of Neurosciences (R.G.M.), Stanford University, CA.
Hiroshi MitsumotoFrom the Department of Neurology (L.H.v.d.B.), Brain Center Rudolf Magnus, University Medical Center Utrecht, the Netherlands; Department of Neurology (E.S.), Mayo Clinic, Rochester, MN; Department of Neurology (G.G.), University of Kansas Medical Center, Kansas City; Department of Medicine (E.A.M.), Massachusetts General Hospital, Biostatistics Center, Harvard Medical School, Boston; Department of Neurology (J.A., H.M.), Columbia University, Eleanor and Lou Gehrig ALS Center, New York, NY; Department of Neurology (R.H.B.), Cedars-Sinai Medical Center, Los Angeles, CA; Department of Neurology (M.B.), University of Miami, FL; Neurological Clinical Research Institute (J.D.B.), Massachusetts General Hospital, Boston; Rita Levi Montalcini Department of Neuroscience (A.C.), University of Torino, Italy; Centre Constitutif SLA (P.C.), Université de Tours, France; Department of Neurology (A.G.), Clinical Research Unit, Montreal Neurological Institute, Neurosurgery, McGill University, Montreal, Canada; National Institute of Neurological Disorders and Stroke (A.K.G.), National Institutes of Health, Bethesda, MD; ALS Center (C.L.-H.), University of California San Francisco; Department of Neuroscience (C.J.M.), Sheffield Institute for Translational Neuroscience, University of Sheffield, UK; Department of Neurology (E.P.P.), Section of ALS & Related Disorders, Cleveland Clinic, OH; Department of Neurology (J.R.), The Center for Restorative Neurology, Loma Linda University School of Medicine, CA; Department of Neurology and Laboratory of Neuroscience (V.S.), Istituto Auxologico Italiano, IRCCS, Milan; Department of Pathophysiology and Transplantation (V.S.), "Dino Ferrari" Centre, Università degli Studi di Milano, Milan, Italy; Nuffield Department of Clinical Neurosciences (M.R.T.), University of Oxford, UK; Neuromuscular Diseases Unit/ALS Clinic (M.W.), Kantonsspital St. Gallen, Switzerland; Carolinas Neuromuscular/ALS-MDA Care Center (B.R.B.), Charlotte; Department of Neurology (B.R.B.), Carolinas Medical Center, University of North Carolina School of Medicine, Charlotte; Forbes Norris ALS Treatment and Research Center (R.G.M.), California Pacific Medical Center San Francisco; and Department of Neurosciences (R.G.M.), Stanford University, CA.
Airlie House ALS Clinical Trials Guidelines Group
Cedars-Sinai Medical Center · US
Funding
ALS Clinical Trials GuidelinesU13NS093847 · NINDS · COLUMBIA UNIVERSITY HEALTH SCIENCES · PI MITSUMOTO, HIROSHI · 2015 to 2015
$20kMotor Neurone Disease Association TURNER/OCT18/989-797NINDS NIH HHS U13 NS093847
Abstract
objectiveTo revise the 1999 Airlie House consensus guidelines for the design and implementation of preclinical therapeutic studies and clinical trials in amyotrophic lateral sclerosis (ALS).
methodsA consensus committee comprising 140 key members of the international ALS community (ALS researchers, clinicians, patient representatives, research funding representatives, industry, and regulatory agencies) addressed 9 areas of need within ALS research: (1) preclinical studies; (2) biological and phenotypic heterogeneity; (3) outcome measures; (4) disease-modifying and symptomatic interventions; (5) recruitment and retention; (6) biomarkers; (7) clinical trial phases; (8) beyond traditional trial designs; and (9) statistical considerations. Assigned to 1 of 8 sections, committee members generated a draft set of guidelines based on a "background" of developing a (pre)clinical question and a "rationale" outlining the evidence and expert opinion. Following a 2-day, face-to-face workshop at the Airlie House Conference Center, a modified Delphi process was used to develop draft consensus research guidelines, which were subsequently reviewed and modified based on comments from the public. Statistical experts drafted a separate document of statistical considerations (section 9).
resultsIn this report, we summarize 112 guidelines and their associated backgrounds and rationales. The full list of guidelines, the statistical considerations, and a glossary of terms can be found in data available from Dryad (appendices e-3-e-5, doi.org/10.5061/dryad.32q9q5d). The authors prioritized 15 guidelines with the greatest potential to improve ALS clinical research.
conclusionThe revised Airlie House ALS Clinical Trials Consensus Guidelines should serve to improve clinical trial design and accelerate the development of effective treatments for patients with ALS.
Indexed as
Amyotrophic Lateral SclerosisClinical Trials as TopicGuidelines as TopicResearch DesignBiomarkersDelphi TechniqueHumansOutcome Assessment, Health CarePatient SelectionStatistics as TopicBiomarkers
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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.