Evidence map›Paper›PMID 39591556›Full record

SynthesisNeurology2024

Functional Outcome Measures to Optimize Drug Development in Spinal and Bulbar Muscular Atrophy: Results From a Meta-Analysis of the Global SBMA Dataset.

Spencer B Huggett, Andrew T N Tebbenkamp, Carlo Rinaldi, Dipa Jayaseelan, Luca Zampedri, Lorenzo Blasi, Andrea Fortuna, Abdullah Alqahtani, Angela Kokkinis, Julia Dahlqvist and 18 more

Abstract readMeta-Analysis
PubMed Full text at PMC Publisher
In one paragraph

Synthesis in Neurology, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.

0numbers the graph read from it
0cells of the map it votes in
4citing 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

4 citing papers in PubMed.

  1. Article
  2. Reliability and construct validity of the Italian version of AMAT scale in SBMA subjects.Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology · 2026
    Article
  3. Article
  4. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

28 authors.

Spencer B HuggettFrom the Nido Biosciences (S.B.H., A.T.N.T., V.V.), Inc., Boston, MA; Institute of Developmental and Regenerative Medicine (IDRM) (C.R.), University of Oxford; Department of Neuromuscular Diseases (D.J., L.Z., P.F.), University College of London, United Kingdom; Department of Neurosciences (L.B., A.F., G.S.), Neuromuscular Center, University of Padova, Italy; Neurogenetics Branch (A.A., A.K., C.G.), National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD; Copenhagen Neuromuscular Center (J.D., J.V.), Rigshospitalet, University of Copenhagen, Denmark; Fondazione IRCSS (S.F., E.C., A.B., C.M., D.P.), Istituto Neurologico Carlo Besta Milano, Italy; Department of Neurology (T.K., Y.K., S.Y.), Nagoya University Graduate School of Medicine; Department of Neurology and Department of Clinical Research Education (M. Katsuno), Nagoya University Graduate School of Medicine, Japan; Centro Clinico Nemo Adulti-Fondazione Serena onlus (A.C.), Policlinico Universitario Agostino Gemelli IRCCS; Centro Clinico Nemo Adulti-Fondazione Serena onlus (M.S.), Policlinico Universitario Agostino Gemelli IRCCS, Section of Neurology, Department of Neuroscience, Faculty of Medicine and Surgery, Università Cattolica del Sacro Cuore, Rome, Italy; and Department of Neurology (M. Kang, J.-S.P.), School of Medicine, Kyungpook National University, Chilgok Hospital, Daegu, Korea.
Andrew T N TebbenkampFrom the Nido Biosciences (S.B.H., A.T.N.T., V.V.), Inc., Boston, MA; Institute of Developmental and Regenerative Medicine (IDRM) (C.R.), University of Oxford; Department of Neuromuscular Diseases (D.J., L.Z., P.F.), University College of London, United Kingdom; Department of Neurosciences (L.B., A.F., G.S.), Neuromuscular Center, University of Padova, Italy; Neurogenetics Branch (A.A., A.K., C.G.), National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD; Copenhagen Neuromuscular Center (J.D., J.V.), Rigshospitalet, University of Copenhagen, Denmark; Fondazione IRCSS (S.F., E.C., A.B., C.M., D.P.), Istituto Neurologico Carlo Besta Milano, Italy; Department of Neurology (T.K., Y.K., S.Y.), Nagoya University Graduate School of Medicine; Department of Neurology and Department of Clinical Research Education (M. Katsuno), Nagoya University Graduate School of Medicine, Japan; Centro Clinico Nemo Adulti-Fondazione Serena onlus (A.C.), Policlinico Universitario Agostino Gemelli IRCCS; Centro Clinico Nemo Adulti-Fondazione Serena onlus (M.S.), Policlinico Universitario Agostino Gemelli IRCCS, Section of Neurology, Department of Neuroscience, Faculty of Medicine and Surgery, Università Cattolica del Sacro Cuore, Rome, Italy; and Department of Neurology (M. Kang, J.-S.P.), School of Medicine, Kyungpook National University, Chilgok Hospital, Daegu, Korea.
Carlo RinaldiFrom the Nido Biosciences (S.B.H., A.T.N.T., V.V.), Inc., Boston, MA; Institute of Developmental and Regenerative Medicine (IDRM) (C.R.), University of Oxford; Department of Neuromuscular Diseases (D.J., L.Z., P.F.), University College of London, United Kingdom; Department of Neurosciences (L.B., A.F., G.S.), Neuromuscular Center, University of Padova, Italy; Neurogenetics Branch (A.A., A.K., C.G.), National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD; Copenhagen Neuromuscular Center (J.D., J.V.), Rigshospitalet, University of Copenhagen, Denmark; Fondazione IRCSS (S.F., E.C., A.B., C.M., D.P.), Istituto Neurologico Carlo Besta Milano, Italy; Department of Neurology (T.K., Y.K., S.Y.), Nagoya University Graduate School of Medicine; Department of Neurology and Department of Clinical Research Education (M. Katsuno), Nagoya University Graduate School of Medicine, Japan; Centro Clinico Nemo Adulti-Fondazione Serena onlus (A.C.), Policlinico Universitario Agostino Gemelli IRCCS; Centro Clinico Nemo Adulti-Fondazione Serena onlus (M.S.), Policlinico Universitario Agostino Gemelli IRCCS, Section of Neurology, Department of Neuroscience, Faculty of Medicine and Surgery, Università Cattolica del Sacro Cuore, Rome, Italy; and Department of Neurology (M. Kang, J.-S.P.), School of Medicine, Kyungpook National University, Chilgok Hospital, Daegu, Korea.
Dipa JayaseelanFrom the Nido Biosciences (S.B.H., A.T.N.T., V.V.), Inc., Boston, MA; Institute of Developmental and Regenerative Medicine (IDRM) (C.R.), University of Oxford; Department of Neuromuscular Diseases (D.J., L.Z., P.F.), University College of London, United Kingdom; Department of Neurosciences (L.B., A.F., G.S.), Neuromuscular Center, University of Padova, Italy; Neurogenetics Branch (A.A., A.K., C.G.), National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD; Copenhagen Neuromuscular Center (J.D., J.V.), Rigshospitalet, University of Copenhagen, Denmark; Fondazione IRCSS (S.F., E.C., A.B., C.M., D.P.), Istituto Neurologico Carlo Besta Milano, Italy; Department of Neurology (T.K., Y.K., S.Y.), Nagoya University Graduate School of Medicine; Department of Neurology and Department of Clinical Research Education (M. Katsuno), Nagoya University Graduate School of Medicine, Japan; Centro Clinico Nemo Adulti-Fondazione Serena onlus (A.C.), Policlinico Universitario Agostino Gemelli IRCCS; Centro Clinico Nemo Adulti-Fondazione Serena onlus (M.S.), Policlinico Universitario Agostino Gemelli IRCCS, Section of Neurology, Department of Neuroscience, Faculty of Medicine and Surgery, Università Cattolica del Sacro Cuore, Rome, Italy; and Department of Neurology (M. Kang, J.-S.P.), School of Medicine, Kyungpook National University, Chilgok Hospital, Daegu, Korea.
Luca ZampedriFrom the Nido Biosciences (S.B.H., A.T.N.T., V.V.), Inc., Boston, MA; Institute of Developmental and Regenerative Medicine (IDRM) (C.R.), University of Oxford; Department of Neuromuscular Diseases (D.J., L.Z., P.F.), University College of London, United Kingdom; Department of Neurosciences (L.B., A.F., G.S.), Neuromuscular Center, University of Padova, Italy; Neurogenetics Branch (A.A., A.K., C.G.), National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD; Copenhagen Neuromuscular Center (J.D., J.V.), Rigshospitalet, University of Copenhagen, Denmark; Fondazione IRCSS (S.F., E.C., A.B., C.M., D.P.), Istituto Neurologico Carlo Besta Milano, Italy; Department of Neurology (T.K., Y.K., S.Y.), Nagoya University Graduate School of Medicine; Department of Neurology and Department of Clinical Research Education (M. Katsuno), Nagoya University Graduate School of Medicine, Japan; Centro Clinico Nemo Adulti-Fondazione Serena onlus (A.C.), Policlinico Universitario Agostino Gemelli IRCCS; Centro Clinico Nemo Adulti-Fondazione Serena onlus (M.S.), Policlinico Universitario Agostino Gemelli IRCCS, Section of Neurology, Department of Neuroscience, Faculty of Medicine and Surgery, Università Cattolica del Sacro Cuore, Rome, Italy; and Department of Neurology (M. Kang, J.-S.P.), School of Medicine, Kyungpook National University, Chilgok Hospital, Daegu, Korea.
Lorenzo BlasiFrom the Nido Biosciences (S.B.H., A.T.N.T., V.V.), Inc., Boston, MA; Institute of Developmental and Regenerative Medicine (IDRM) (C.R.), University of Oxford; Department of Neuromuscular Diseases (D.J., L.Z., P.F.), University College of London, United Kingdom; Department of Neurosciences (L.B., A.F., G.S.), Neuromuscular Center, University of Padova, Italy; Neurogenetics Branch (A.A., A.K., C.G.), National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD; Copenhagen Neuromuscular Center (J.D., J.V.), Rigshospitalet, University of Copenhagen, Denmark; Fondazione IRCSS (S.F., E.C., A.B., C.M., D.P.), Istituto Neurologico Carlo Besta Milano, Italy; Department of Neurology (T.K., Y.K., S.Y.), Nagoya University Graduate School of Medicine; Department of Neurology and Department of Clinical Research Education (M. Katsuno), Nagoya University Graduate School of Medicine, Japan; Centro Clinico Nemo Adulti-Fondazione Serena onlus (A.C.), Policlinico Universitario Agostino Gemelli IRCCS; Centro Clinico Nemo Adulti-Fondazione Serena onlus (M.S.), Policlinico Universitario Agostino Gemelli IRCCS, Section of Neurology, Department of Neuroscience, Faculty of Medicine and Surgery, Università Cattolica del Sacro Cuore, Rome, Italy; and Department of Neurology (M. Kang, J.-S.P.), School of Medicine, Kyungpook National University, Chilgok Hospital, Daegu, Korea.
Andrea FortunaFrom the Nido Biosciences (S.B.H., A.T.N.T., V.V.), Inc., Boston, MA; Institute of Developmental and Regenerative Medicine (IDRM) (C.R.), University of Oxford; Department of Neuromuscular Diseases (D.J., L.Z., P.F.), University College of London, United Kingdom; Department of Neurosciences (L.B., A.F., G.S.), Neuromuscular Center, University of Padova, Italy; Neurogenetics Branch (A.A., A.K., C.G.), National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD; Copenhagen Neuromuscular Center (J.D., J.V.), Rigshospitalet, University of Copenhagen, Denmark; Fondazione IRCSS (S.F., E.C., A.B., C.M., D.P.), Istituto Neurologico Carlo Besta Milano, Italy; Department of Neurology (T.K., Y.K., S.Y.), Nagoya University Graduate School of Medicine; Department of Neurology and Department of Clinical Research Education (M. Katsuno), Nagoya University Graduate School of Medicine, Japan; Centro Clinico Nemo Adulti-Fondazione Serena onlus (A.C.), Policlinico Universitario Agostino Gemelli IRCCS; Centro Clinico Nemo Adulti-Fondazione Serena onlus (M.S.), Policlinico Universitario Agostino Gemelli IRCCS, Section of Neurology, Department of Neuroscience, Faculty of Medicine and Surgery, Università Cattolica del Sacro Cuore, Rome, Italy; and Department of Neurology (M. Kang, J.-S.P.), School of Medicine, Kyungpook National University, Chilgok Hospital, Daegu, Korea.
Abdullah AlqahtaniFrom the Nido Biosciences (S.B.H., A.T.N.T., V.V.), Inc., Boston, MA; Institute of Developmental and Regenerative Medicine (IDRM) (C.R.), University of Oxford; Department of Neuromuscular Diseases (D.J., L.Z., P.F.), University College of London, United Kingdom; Department of Neurosciences (L.B., A.F., G.S.), Neuromuscular Center, University of Padova, Italy; Neurogenetics Branch (A.A., A.K., C.G.), National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD; Copenhagen Neuromuscular Center (J.D., J.V.), Rigshospitalet, University of Copenhagen, Denmark; Fondazione IRCSS (S.F., E.C., A.B., C.M., D.P.), Istituto Neurologico Carlo Besta Milano, Italy; Department of Neurology (T.K., Y.K., S.Y.), Nagoya University Graduate School of Medicine; Department of Neurology and Department of Clinical Research Education (M. Katsuno), Nagoya University Graduate School of Medicine, Japan; Centro Clinico Nemo Adulti-Fondazione Serena onlus (A.C.), Policlinico Universitario Agostino Gemelli IRCCS; Centro Clinico Nemo Adulti-Fondazione Serena onlus (M.S.), Policlinico Universitario Agostino Gemelli IRCCS, Section of Neurology, Department of Neuroscience, Faculty of Medicine and Surgery, Università Cattolica del Sacro Cuore, Rome, Italy; and Department of Neurology (M. Kang, J.-S.P.), School of Medicine, Kyungpook National University, Chilgok Hospital, Daegu, Korea.ORCID 0009-0008-8924-1543
Angela KokkinisFrom the Nido Biosciences (S.B.H., A.T.N.T., V.V.), Inc., Boston, MA; Institute of Developmental and Regenerative Medicine (IDRM) (C.R.), University of Oxford; Department of Neuromuscular Diseases (D.J., L.Z., P.F.), University College of London, United Kingdom; Department of Neurosciences (L.B., A.F., G.S.), Neuromuscular Center, University of Padova, Italy; Neurogenetics Branch (A.A., A.K., C.G.), National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD; Copenhagen Neuromuscular Center (J.D., J.V.), Rigshospitalet, University of Copenhagen, Denmark; Fondazione IRCSS (S.F., E.C., A.B., C.M., D.P.), Istituto Neurologico Carlo Besta Milano, Italy; Department of Neurology (T.K., Y.K., S.Y.), Nagoya University Graduate School of Medicine; Department of Neurology and Department of Clinical Research Education (M. Katsuno), Nagoya University Graduate School of Medicine, Japan; Centro Clinico Nemo Adulti-Fondazione Serena onlus (A.C.), Policlinico Universitario Agostino Gemelli IRCCS; Centro Clinico Nemo Adulti-Fondazione Serena onlus (M.S.), Policlinico Universitario Agostino Gemelli IRCCS, Section of Neurology, Department of Neuroscience, Faculty of Medicine and Surgery, Università Cattolica del Sacro Cuore, Rome, Italy; and Department of Neurology (M. Kang, J.-S.P.), School of Medicine, Kyungpook National University, Chilgok Hospital, Daegu, Korea.ORCID 0009-0001-5683-1415
Julia DahlqvistFrom the Nido Biosciences (S.B.H., A.T.N.T., V.V.), Inc., Boston, MA; Institute of Developmental and Regenerative Medicine (IDRM) (C.R.), University of Oxford; Department of Neuromuscular Diseases (D.J., L.Z., P.F.), University College of London, United Kingdom; Department of Neurosciences (L.B., A.F., G.S.), Neuromuscular Center, University of Padova, Italy; Neurogenetics Branch (A.A., A.K., C.G.), National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD; Copenhagen Neuromuscular Center (J.D., J.V.), Rigshospitalet, University of Copenhagen, Denmark; Fondazione IRCSS (S.F., E.C., A.B., C.M., D.P.), Istituto Neurologico Carlo Besta Milano, Italy; Department of Neurology (T.K., Y.K., S.Y.), Nagoya University Graduate School of Medicine; Department of Neurology and Department of Clinical Research Education (M. Katsuno), Nagoya University Graduate School of Medicine, Japan; Centro Clinico Nemo Adulti-Fondazione Serena onlus (A.C.), Policlinico Universitario Agostino Gemelli IRCCS; Centro Clinico Nemo Adulti-Fondazione Serena onlus (M.S.), Policlinico Universitario Agostino Gemelli IRCCS, Section of Neurology, Department of Neuroscience, Faculty of Medicine and Surgery, Università Cattolica del Sacro Cuore, Rome, Italy; and Department of Neurology (M. Kang, J.-S.P.), School of Medicine, Kyungpook National University, Chilgok Hospital, Daegu, Korea.
Silvia FenuFrom the Nido Biosciences (S.B.H., A.T.N.T., V.V.), Inc., Boston, MA; Institute of Developmental and Regenerative Medicine (IDRM) (C.R.), University of Oxford; Department of Neuromuscular Diseases (D.J., L.Z., P.F.), University College of London, United Kingdom; Department of Neurosciences (L.B., A.F., G.S.), Neuromuscular Center, University of Padova, Italy; Neurogenetics Branch (A.A., A.K., C.G.), National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD; Copenhagen Neuromuscular Center (J.D., J.V.), Rigshospitalet, University of Copenhagen, Denmark; Fondazione IRCSS (S.F., E.C., A.B., C.M., D.P.), Istituto Neurologico Carlo Besta Milano, Italy; Department of Neurology (T.K., Y.K., S.Y.), Nagoya University Graduate School of Medicine; Department of Neurology and Department of Clinical Research Education (M. Katsuno), Nagoya University Graduate School of Medicine, Japan; Centro Clinico Nemo Adulti-Fondazione Serena onlus (A.C.), Policlinico Universitario Agostino Gemelli IRCCS; Centro Clinico Nemo Adulti-Fondazione Serena onlus (M.S.), Policlinico Universitario Agostino Gemelli IRCCS, Section of Neurology, Department of Neuroscience, Faculty of Medicine and Surgery, Università Cattolica del Sacro Cuore, Rome, Italy; and Department of Neurology (M. Kang, J.-S.P.), School of Medicine, Kyungpook National University, Chilgok Hospital, Daegu, Korea.ORCID 0000-0002-5233-6580
Eleonora CavalcaFrom the Nido Biosciences (S.B.H., A.T.N.T., V.V.), Inc., Boston, MA; Institute of Developmental and Regenerative Medicine (IDRM) (C.R.), University of Oxford; Department of Neuromuscular Diseases (D.J., L.Z., P.F.), University College of London, United Kingdom; Department of Neurosciences (L.B., A.F., G.S.), Neuromuscular Center, University of Padova, Italy; Neurogenetics Branch (A.A., A.K., C.G.), National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD; Copenhagen Neuromuscular Center (J.D., J.V.), Rigshospitalet, University of Copenhagen, Denmark; Fondazione IRCSS (S.F., E.C., A.B., C.M., D.P.), Istituto Neurologico Carlo Besta Milano, Italy; Department of Neurology (T.K., Y.K., S.Y.), Nagoya University Graduate School of Medicine; Department of Neurology and Department of Clinical Research Education (M. Katsuno), Nagoya University Graduate School of Medicine, Japan; Centro Clinico Nemo Adulti-Fondazione Serena onlus (A.C.), Policlinico Universitario Agostino Gemelli IRCCS; Centro Clinico Nemo Adulti-Fondazione Serena onlus (M.S.), Policlinico Universitario Agostino Gemelli IRCCS, Section of Neurology, Department of Neuroscience, Faculty of Medicine and Surgery, Università Cattolica del Sacro Cuore, Rome, Italy; and Department of Neurology (M. Kang, J.-S.P.), School of Medicine, Kyungpook National University, Chilgok Hospital, Daegu, Korea.
Alessandro BertiniFrom the Nido Biosciences (S.B.H., A.T.N.T., V.V.), Inc., Boston, MA; Institute of Developmental and Regenerative Medicine (IDRM) (C.R.), University of Oxford; Department of Neuromuscular Diseases (D.J., L.Z., P.F.), University College of London, United Kingdom; Department of Neurosciences (L.B., A.F., G.S.), Neuromuscular Center, University of Padova, Italy; Neurogenetics Branch (A.A., A.K., C.G.), National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD; Copenhagen Neuromuscular Center (J.D., J.V.), Rigshospitalet, University of Copenhagen, Denmark; Fondazione IRCSS (S.F., E.C., A.B., C.M., D.P.), Istituto Neurologico Carlo Besta Milano, Italy; Department of Neurology (T.K., Y.K., S.Y.), Nagoya University Graduate School of Medicine; Department of Neurology and Department of Clinical Research Education (M. Katsuno), Nagoya University Graduate School of Medicine, Japan; Centro Clinico Nemo Adulti-Fondazione Serena onlus (A.C.), Policlinico Universitario Agostino Gemelli IRCCS; Centro Clinico Nemo Adulti-Fondazione Serena onlus (M.S.), Policlinico Universitario Agostino Gemelli IRCCS, Section of Neurology, Department of Neuroscience, Faculty of Medicine and Surgery, Università Cattolica del Sacro Cuore, Rome, Italy; and Department of Neurology (M. Kang, J.-S.P.), School of Medicine, Kyungpook National University, Chilgok Hospital, Daegu, Korea.
Caterina MariottiFrom the Nido Biosciences (S.B.H., A.T.N.T., V.V.), Inc., Boston, MA; Institute of Developmental and Regenerative Medicine (IDRM) (C.R.), University of Oxford; Department of Neuromuscular Diseases (D.J., L.Z., P.F.), University College of London, United Kingdom; Department of Neurosciences (L.B., A.F., G.S.), Neuromuscular Center, University of Padova, Italy; Neurogenetics Branch (A.A., A.K., C.G.), National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD; Copenhagen Neuromuscular Center (J.D., J.V.), Rigshospitalet, University of Copenhagen, Denmark; Fondazione IRCSS (S.F., E.C., A.B., C.M., D.P.), Istituto Neurologico Carlo Besta Milano, Italy; Department of Neurology (T.K., Y.K., S.Y.), Nagoya University Graduate School of Medicine; Department of Neurology and Department of Clinical Research Education (M. Katsuno), Nagoya University Graduate School of Medicine, Japan; Centro Clinico Nemo Adulti-Fondazione Serena onlus (A.C.), Policlinico Universitario Agostino Gemelli IRCCS; Centro Clinico Nemo Adulti-Fondazione Serena onlus (M.S.), Policlinico Universitario Agostino Gemelli IRCCS, Section of Neurology, Department of Neuroscience, Faculty of Medicine and Surgery, Università Cattolica del Sacro Cuore, Rome, Italy; and Department of Neurology (M. Kang, J.-S.P.), School of Medicine, Kyungpook National University, Chilgok Hospital, Daegu, Korea.ORCID 0000-0003-2405-3564
Christopher GrunseichFrom the Nido Biosciences (S.B.H., A.T.N.T., V.V.), Inc., Boston, MA; Institute of Developmental and Regenerative Medicine (IDRM) (C.R.), University of Oxford; Department of Neuromuscular Diseases (D.J., L.Z., P.F.), University College of London, United Kingdom; Department of Neurosciences (L.B., A.F., G.S.), Neuromuscular Center, University of Padova, Italy; Neurogenetics Branch (A.A., A.K., C.G.), National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD; Copenhagen Neuromuscular Center (J.D., J.V.), Rigshospitalet, University of Copenhagen, Denmark; Fondazione IRCSS (S.F., E.C., A.B., C.M., D.P.), Istituto Neurologico Carlo Besta Milano, Italy; Department of Neurology (T.K., Y.K., S.Y.), Nagoya University Graduate School of Medicine; Department of Neurology and Department of Clinical Research Education (M. Katsuno), Nagoya University Graduate School of Medicine, Japan; Centro Clinico Nemo Adulti-Fondazione Serena onlus (A.C.), Policlinico Universitario Agostino Gemelli IRCCS; Centro Clinico Nemo Adulti-Fondazione Serena onlus (M.S.), Policlinico Universitario Agostino Gemelli IRCCS, Section of Neurology, Department of Neuroscience, Faculty of Medicine and Surgery, Università Cattolica del Sacro Cuore, Rome, Italy; and Department of Neurology (M. Kang, J.-S.P.), School of Medicine, Kyungpook National University, Chilgok Hospital, Daegu, Korea.ORCID 0000-0003-4994-2472
Takahiro KawaseFrom the Nido Biosciences (S.B.H., A.T.N.T., V.V.), Inc., Boston, MA; Institute of Developmental and Regenerative Medicine (IDRM) (C.R.), University of Oxford; Department of Neuromuscular Diseases (D.J., L.Z., P.F.), University College of London, United Kingdom; Department of Neurosciences (L.B., A.F., G.S.), Neuromuscular Center, University of Padova, Italy; Neurogenetics Branch (A.A., A.K., C.G.), National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD; Copenhagen Neuromuscular Center (J.D., J.V.), Rigshospitalet, University of Copenhagen, Denmark; Fondazione IRCSS (S.F., E.C., A.B., C.M., D.P.), Istituto Neurologico Carlo Besta Milano, Italy; Department of Neurology (T.K., Y.K., S.Y.), Nagoya University Graduate School of Medicine; Department of Neurology and Department of Clinical Research Education (M. Katsuno), Nagoya University Graduate School of Medicine, Japan; Centro Clinico Nemo Adulti-Fondazione Serena onlus (A.C.), Policlinico Universitario Agostino Gemelli IRCCS; Centro Clinico Nemo Adulti-Fondazione Serena onlus (M.S.), Policlinico Universitario Agostino Gemelli IRCCS, Section of Neurology, Department of Neuroscience, Faculty of Medicine and Surgery, Università Cattolica del Sacro Cuore, Rome, Italy; and Department of Neurology (M. Kang, J.-S.P.), School of Medicine, Kyungpook National University, Chilgok Hospital, Daegu, Korea.
Yoshiyuki KishimotoFrom the Nido Biosciences (S.B.H., A.T.N.T., V.V.), Inc., Boston, MA; Institute of Developmental and Regenerative Medicine (IDRM) (C.R.), University of Oxford; Department of Neuromuscular Diseases (D.J., L.Z., P.F.), University College of London, United Kingdom; Department of Neurosciences (L.B., A.F., G.S.), Neuromuscular Center, University of Padova, Italy; Neurogenetics Branch (A.A., A.K., C.G.), National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD; Copenhagen Neuromuscular Center (J.D., J.V.), Rigshospitalet, University of Copenhagen, Denmark; Fondazione IRCSS (S.F., E.C., A.B., C.M., D.P.), Istituto Neurologico Carlo Besta Milano, Italy; Department of Neurology (T.K., Y.K., S.Y.), Nagoya University Graduate School of Medicine; Department of Neurology and Department of Clinical Research Education (M. Katsuno), Nagoya University Graduate School of Medicine, Japan; Centro Clinico Nemo Adulti-Fondazione Serena onlus (A.C.), Policlinico Universitario Agostino Gemelli IRCCS; Centro Clinico Nemo Adulti-Fondazione Serena onlus (M.S.), Policlinico Universitario Agostino Gemelli IRCCS, Section of Neurology, Department of Neuroscience, Faculty of Medicine and Surgery, Università Cattolica del Sacro Cuore, Rome, Italy; and Department of Neurology (M. Kang, J.-S.P.), School of Medicine, Kyungpook National University, Chilgok Hospital, Daegu, Korea.
Shinichiro YamadaFrom the Nido Biosciences (S.B.H., A.T.N.T., V.V.), Inc., Boston, MA; Institute of Developmental and Regenerative Medicine (IDRM) (C.R.), University of Oxford; Department of Neuromuscular Diseases (D.J., L.Z., P.F.), University College of London, United Kingdom; Department of Neurosciences (L.B., A.F., G.S.), Neuromuscular Center, University of Padova, Italy; Neurogenetics Branch (A.A., A.K., C.G.), National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD; Copenhagen Neuromuscular Center (J.D., J.V.), Rigshospitalet, University of Copenhagen, Denmark; Fondazione IRCSS (S.F., E.C., A.B., C.M., D.P.), Istituto Neurologico Carlo Besta Milano, Italy; Department of Neurology (T.K., Y.K., S.Y.), Nagoya University Graduate School of Medicine; Department of Neurology and Department of Clinical Research Education (M. Katsuno), Nagoya University Graduate School of Medicine, Japan; Centro Clinico Nemo Adulti-Fondazione Serena onlus (A.C.), Policlinico Universitario Agostino Gemelli IRCCS; Centro Clinico Nemo Adulti-Fondazione Serena onlus (M.S.), Policlinico Universitario Agostino Gemelli IRCCS, Section of Neurology, Department of Neuroscience, Faculty of Medicine and Surgery, Università Cattolica del Sacro Cuore, Rome, Italy; and Department of Neurology (M. Kang, J.-S.P.), School of Medicine, Kyungpook National University, Chilgok Hospital, Daegu, Korea.
Masahisa KatsunoFrom the Nido Biosciences (S.B.H., A.T.N.T., V.V.), Inc., Boston, MA; Institute of Developmental and Regenerative Medicine (IDRM) (C.R.), University of Oxford; Department of Neuromuscular Diseases (D.J., L.Z., P.F.), University College of London, United Kingdom; Department of Neurosciences (L.B., A.F., G.S.), Neuromuscular Center, University of Padova, Italy; Neurogenetics Branch (A.A., A.K., C.G.), National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD; Copenhagen Neuromuscular Center (J.D., J.V.), Rigshospitalet, University of Copenhagen, Denmark; Fondazione IRCSS (S.F., E.C., A.B., C.M., D.P.), Istituto Neurologico Carlo Besta Milano, Italy; Department of Neurology (T.K., Y.K., S.Y.), Nagoya University Graduate School of Medicine; Department of Neurology and Department of Clinical Research Education (M. Katsuno), Nagoya University Graduate School of Medicine, Japan; Centro Clinico Nemo Adulti-Fondazione Serena onlus (A.C.), Policlinico Universitario Agostino Gemelli IRCCS; Centro Clinico Nemo Adulti-Fondazione Serena onlus (M.S.), Policlinico Universitario Agostino Gemelli IRCCS, Section of Neurology, Department of Neuroscience, Faculty of Medicine and Surgery, Università Cattolica del Sacro Cuore, Rome, Italy; and Department of Neurology (M. Kang, J.-S.P.), School of Medicine, Kyungpook National University, Chilgok Hospital, Daegu, Korea.ORCID 0000-0001-9453-9311
Pietro FrattaFrom the Nido Biosciences (S.B.H., A.T.N.T., V.V.), Inc., Boston, MA; Institute of Developmental and Regenerative Medicine (IDRM) (C.R.), University of Oxford; Department of Neuromuscular Diseases (D.J., L.Z., P.F.), University College of London, United Kingdom; Department of Neurosciences (L.B., A.F., G.S.), Neuromuscular Center, University of Padova, Italy; Neurogenetics Branch (A.A., A.K., C.G.), National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD; Copenhagen Neuromuscular Center (J.D., J.V.), Rigshospitalet, University of Copenhagen, Denmark; Fondazione IRCSS (S.F., E.C., A.B., C.M., D.P.), Istituto Neurologico Carlo Besta Milano, Italy; Department of Neurology (T.K., Y.K., S.Y.), Nagoya University Graduate School of Medicine; Department of Neurology and Department of Clinical Research Education (M. Katsuno), Nagoya University Graduate School of Medicine, Japan; Centro Clinico Nemo Adulti-Fondazione Serena onlus (A.C.), Policlinico Universitario Agostino Gemelli IRCCS; Centro Clinico Nemo Adulti-Fondazione Serena onlus (M.S.), Policlinico Universitario Agostino Gemelli IRCCS, Section of Neurology, Department of Neuroscience, Faculty of Medicine and Surgery, Università Cattolica del Sacro Cuore, Rome, Italy; and Department of Neurology (M. Kang, J.-S.P.), School of Medicine, Kyungpook National University, Chilgok Hospital, Daegu, Korea.ORCID 0000-0002-7554-1632
Amelia ConteFrom the Nido Biosciences (S.B.H., A.T.N.T., V.V.), Inc., Boston, MA; Institute of Developmental and Regenerative Medicine (IDRM) (C.R.), University of Oxford; Department of Neuromuscular Diseases (D.J., L.Z., P.F.), University College of London, United Kingdom; Department of Neurosciences (L.B., A.F., G.S.), Neuromuscular Center, University of Padova, Italy; Neurogenetics Branch (A.A., A.K., C.G.), National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD; Copenhagen Neuromuscular Center (J.D., J.V.), Rigshospitalet, University of Copenhagen, Denmark; Fondazione IRCSS (S.F., E.C., A.B., C.M., D.P.), Istituto Neurologico Carlo Besta Milano, Italy; Department of Neurology (T.K., Y.K., S.Y.), Nagoya University Graduate School of Medicine; Department of Neurology and Department of Clinical Research Education (M. Katsuno), Nagoya University Graduate School of Medicine, Japan; Centro Clinico Nemo Adulti-Fondazione Serena onlus (A.C.), Policlinico Universitario Agostino Gemelli IRCCS; Centro Clinico Nemo Adulti-Fondazione Serena onlus (M.S.), Policlinico Universitario Agostino Gemelli IRCCS, Section of Neurology, Department of Neuroscience, Faculty of Medicine and Surgery, Università Cattolica del Sacro Cuore, Rome, Italy; and Department of Neurology (M. Kang, J.-S.P.), School of Medicine, Kyungpook National University, Chilgok Hospital, Daegu, Korea.
Mario SabatelliFrom the Nido Biosciences (S.B.H., A.T.N.T., V.V.), Inc., Boston, MA; Institute of Developmental and Regenerative Medicine (IDRM) (C.R.), University of Oxford; Department of Neuromuscular Diseases (D.J., L.Z., P.F.), University College of London, United Kingdom; Department of Neurosciences (L.B., A.F., G.S.), Neuromuscular Center, University of Padova, Italy; Neurogenetics Branch (A.A., A.K., C.G.), National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD; Copenhagen Neuromuscular Center (J.D., J.V.), Rigshospitalet, University of Copenhagen, Denmark; Fondazione IRCSS (S.F., E.C., A.B., C.M., D.P.), Istituto Neurologico Carlo Besta Milano, Italy; Department of Neurology (T.K., Y.K., S.Y.), Nagoya University Graduate School of Medicine; Department of Neurology and Department of Clinical Research Education (M. Katsuno), Nagoya University Graduate School of Medicine, Japan; Centro Clinico Nemo Adulti-Fondazione Serena onlus (A.C.), Policlinico Universitario Agostino Gemelli IRCCS; Centro Clinico Nemo Adulti-Fondazione Serena onlus (M.S.), Policlinico Universitario Agostino Gemelli IRCCS, Section of Neurology, Department of Neuroscience, Faculty of Medicine and Surgery, Università Cattolica del Sacro Cuore, Rome, Italy; and Department of Neurology (M. Kang, J.-S.P.), School of Medicine, Kyungpook National University, Chilgok Hospital, Daegu, Korea.
Gianni SoraruFrom the Nido Biosciences (S.B.H., A.T.N.T., V.V.), Inc., Boston, MA; Institute of Developmental and Regenerative Medicine (IDRM) (C.R.), University of Oxford; Department of Neuromuscular Diseases (D.J., L.Z., P.F.), University College of London, United Kingdom; Department of Neurosciences (L.B., A.F., G.S.), Neuromuscular Center, University of Padova, Italy; Neurogenetics Branch (A.A., A.K., C.G.), National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD; Copenhagen Neuromuscular Center (J.D., J.V.), Rigshospitalet, University of Copenhagen, Denmark; Fondazione IRCSS (S.F., E.C., A.B., C.M., D.P.), Istituto Neurologico Carlo Besta Milano, Italy; Department of Neurology (T.K., Y.K., S.Y.), Nagoya University Graduate School of Medicine; Department of Neurology and Department of Clinical Research Education (M. Katsuno), Nagoya University Graduate School of Medicine, Japan; Centro Clinico Nemo Adulti-Fondazione Serena onlus (A.C.), Policlinico Universitario Agostino Gemelli IRCCS; Centro Clinico Nemo Adulti-Fondazione Serena onlus (M.S.), Policlinico Universitario Agostino Gemelli IRCCS, Section of Neurology, Department of Neuroscience, Faculty of Medicine and Surgery, Università Cattolica del Sacro Cuore, Rome, Italy; and Department of Neurology (M. Kang, J.-S.P.), School of Medicine, Kyungpook National University, Chilgok Hospital, Daegu, Korea.
John VissingFrom the Nido Biosciences (S.B.H., A.T.N.T., V.V.), Inc., Boston, MA; Institute of Developmental and Regenerative Medicine (IDRM) (C.R.), University of Oxford; Department of Neuromuscular Diseases (D.J., L.Z., P.F.), University College of London, United Kingdom; Department of Neurosciences (L.B., A.F., G.S.), Neuromuscular Center, University of Padova, Italy; Neurogenetics Branch (A.A., A.K., C.G.), National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD; Copenhagen Neuromuscular Center (J.D., J.V.), Rigshospitalet, University of Copenhagen, Denmark; Fondazione IRCSS (S.F., E.C., A.B., C.M., D.P.), Istituto Neurologico Carlo Besta Milano, Italy; Department of Neurology (T.K., Y.K., S.Y.), Nagoya University Graduate School of Medicine; Department of Neurology and Department of Clinical Research Education (M. Katsuno), Nagoya University Graduate School of Medicine, Japan; Centro Clinico Nemo Adulti-Fondazione Serena onlus (A.C.), Policlinico Universitario Agostino Gemelli IRCCS; Centro Clinico Nemo Adulti-Fondazione Serena onlus (M.S.), Policlinico Universitario Agostino Gemelli IRCCS, Section of Neurology, Department of Neuroscience, Faculty of Medicine and Surgery, Università Cattolica del Sacro Cuore, Rome, Italy; and Department of Neurology (M. Kang, J.-S.P.), School of Medicine, Kyungpook National University, Chilgok Hospital, Daegu, Korea.ORCID 0000-0001-6144-8544
Minsung KangFrom the Nido Biosciences (S.B.H., A.T.N.T., V.V.), Inc., Boston, MA; Institute of Developmental and Regenerative Medicine (IDRM) (C.R.), University of Oxford; Department of Neuromuscular Diseases (D.J., L.Z., P.F.), University College of London, United Kingdom; Department of Neurosciences (L.B., A.F., G.S.), Neuromuscular Center, University of Padova, Italy; Neurogenetics Branch (A.A., A.K., C.G.), National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD; Copenhagen Neuromuscular Center (J.D., J.V.), Rigshospitalet, University of Copenhagen, Denmark; Fondazione IRCSS (S.F., E.C., A.B., C.M., D.P.), Istituto Neurologico Carlo Besta Milano, Italy; Department of Neurology (T.K., Y.K., S.Y.), Nagoya University Graduate School of Medicine; Department of Neurology and Department of Clinical Research Education (M. Katsuno), Nagoya University Graduate School of Medicine, Japan; Centro Clinico Nemo Adulti-Fondazione Serena onlus (A.C.), Policlinico Universitario Agostino Gemelli IRCCS; Centro Clinico Nemo Adulti-Fondazione Serena onlus (M.S.), Policlinico Universitario Agostino Gemelli IRCCS, Section of Neurology, Department of Neuroscience, Faculty of Medicine and Surgery, Università Cattolica del Sacro Cuore, Rome, Italy; and Department of Neurology (M. Kang, J.-S.P.), School of Medicine, Kyungpook National University, Chilgok Hospital, Daegu, Korea.
Jin-Sung ParkFrom the Nido Biosciences (S.B.H., A.T.N.T., V.V.), Inc., Boston, MA; Institute of Developmental and Regenerative Medicine (IDRM) (C.R.), University of Oxford; Department of Neuromuscular Diseases (D.J., L.Z., P.F.), University College of London, United Kingdom; Department of Neurosciences (L.B., A.F., G.S.), Neuromuscular Center, University of Padova, Italy; Neurogenetics Branch (A.A., A.K., C.G.), National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD; Copenhagen Neuromuscular Center (J.D., J.V.), Rigshospitalet, University of Copenhagen, Denmark; Fondazione IRCSS (S.F., E.C., A.B., C.M., D.P.), Istituto Neurologico Carlo Besta Milano, Italy; Department of Neurology (T.K., Y.K., S.Y.), Nagoya University Graduate School of Medicine; Department of Neurology and Department of Clinical Research Education (M. Katsuno), Nagoya University Graduate School of Medicine, Japan; Centro Clinico Nemo Adulti-Fondazione Serena onlus (A.C.), Policlinico Universitario Agostino Gemelli IRCCS; Centro Clinico Nemo Adulti-Fondazione Serena onlus (M.S.), Policlinico Universitario Agostino Gemelli IRCCS, Section of Neurology, Department of Neuroscience, Faculty of Medicine and Surgery, Università Cattolica del Sacro Cuore, Rome, Italy; and Department of Neurology (M. Kang, J.-S.P.), School of Medicine, Kyungpook National University, Chilgok Hospital, Daegu, Korea.
Davide PareysonFrom the Nido Biosciences (S.B.H., A.T.N.T., V.V.), Inc., Boston, MA; Institute of Developmental and Regenerative Medicine (IDRM) (C.R.), University of Oxford; Department of Neuromuscular Diseases (D.J., L.Z., P.F.), University College of London, United Kingdom; Department of Neurosciences (L.B., A.F., G.S.), Neuromuscular Center, University of Padova, Italy; Neurogenetics Branch (A.A., A.K., C.G.), National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD; Copenhagen Neuromuscular Center (J.D., J.V.), Rigshospitalet, University of Copenhagen, Denmark; Fondazione IRCSS (S.F., E.C., A.B., C.M., D.P.), Istituto Neurologico Carlo Besta Milano, Italy; Department of Neurology (T.K., Y.K., S.Y.), Nagoya University Graduate School of Medicine; Department of Neurology and Department of Clinical Research Education (M. Katsuno), Nagoya University Graduate School of Medicine, Japan; Centro Clinico Nemo Adulti-Fondazione Serena onlus (A.C.), Policlinico Universitario Agostino Gemelli IRCCS; Centro Clinico Nemo Adulti-Fondazione Serena onlus (M.S.), Policlinico Universitario Agostino Gemelli IRCCS, Section of Neurology, Department of Neuroscience, Faculty of Medicine and Surgery, Università Cattolica del Sacro Cuore, Rome, Italy; and Department of Neurology (M. Kang, J.-S.P.), School of Medicine, Kyungpook National University, Chilgok Hospital, Daegu, Korea.ORCID 0000-0001-6854-765X
Vissia VigliettaFrom the Nido Biosciences (S.B.H., A.T.N.T., V.V.), Inc., Boston, MA; Institute of Developmental and Regenerative Medicine (IDRM) (C.R.), University of Oxford; Department of Neuromuscular Diseases (D.J., L.Z., P.F.), University College of London, United Kingdom; Department of Neurosciences (L.B., A.F., G.S.), Neuromuscular Center, University of Padova, Italy; Neurogenetics Branch (A.A., A.K., C.G.), National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD; Copenhagen Neuromuscular Center (J.D., J.V.), Rigshospitalet, University of Copenhagen, Denmark; Fondazione IRCSS (S.F., E.C., A.B., C.M., D.P.), Istituto Neurologico Carlo Besta Milano, Italy; Department of Neurology (T.K., Y.K., S.Y.), Nagoya University Graduate School of Medicine; Department of Neurology and Department of Clinical Research Education (M. Katsuno), Nagoya University Graduate School of Medicine, Japan; Centro Clinico Nemo Adulti-Fondazione Serena onlus (A.C.), Policlinico Universitario Agostino Gemelli IRCCS; Centro Clinico Nemo Adulti-Fondazione Serena onlus (M.S.), Policlinico Universitario Agostino Gemelli IRCCS, Section of Neurology, Department of Neuroscience, Faculty of Medicine and Surgery, Università Cattolica del Sacro Cuore, Rome, Italy; and Department of Neurology (M. Kang, J.-S.P.), School of Medicine, Kyungpook National University, Chilgok Hospital, Daegu, Korea.ORCID 0009-0005-6270-0904

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

BACKGROUND AND

objectivesSpinal and bulbar muscular atrophy (SBMA) is a rare, slowly progressive, and debilitating disease without effective treatments available. Lack of reliable biomarkers and sensitive outcome measures makes clinical research conduct challenging. The primary objective of this study was to identify clinically meaningful and statistically sensitive outcome measures enabling the evaluation of therapeutic interventions in late-stage clinical trials.

methodsThis study was a meta-analysis of SBMA patient-level data from 6 observational studies conducted in Italy, South Korea, Denmark, United Kingdom, Japan, and United States. Patients with confirmed SBMA genetic diagnosis and differing severity were enrolled following individual site protocols. Routine assessments were performed longitudinally for approximately 3 years, including one or more clinical outcomes, such as SBMA functional rating scale (SBMAFRS), 6-minute walk test (6MWT), quantitative muscle testing (QMT), and Adult Myopathy Assessment Tool (AMAT). A modified scale, m-SBMAFRS, was derived by including only lower limb and trunk subscales having lower variability and larger effect size compared with the others. Changes from baseline at follow-up time points were calculated for all measures, and percent changes using random slope models were calculated to compare clinical measure performances. A survey conducted on 196 patients by the Coordination of Rare Diseases at Sanford (CoRDS), elucidating the impact of specific disease aspects on patients' lives, was also evaluated to corroborate these research outcomes.

resultsThis global SBMA dataset analyzed data from 278 men (mean age = 59.7 ± 10.8 years, mean disease duration = 17.7 ± 11.9 years). Patients progressed on SBMAFRS (-4.7 ± 6.2 points after 38 months with 1-year standard response mean [SRM] = 0.6) and 6MWT (distance walked decreased by -53.2 ± 87.0 meters after 26 months with 1-year SRM = 0.5). These measures showed lower variability and larger effect size than AMAT and QMT (1-year SRM = 0.1 and -0.2, respectively) and confirmed SBMA linear progression across a range of disease stages. The m-SBMAFRS also showed a significant yearly decline of 0.9 ± 1.5 points (SRM = 0.6) and more consistent performance with less variability across clinical sites. The CoRDS survey confirmed the relevance of lower limb strength and mobility, which correlated with higher quality-of-life metrics and were reported by patients as predominant disease issues. DISCUSSION: We generated a comprehensive global SBMA dataset, enabling the identification of sensitive functional end points for clinical trials. Possible limitations relate to data collection nuances across sites that a single study protocol could override.

Indexed as

Outcome Assessment, Health CareAgedBulbo-Spinal Atrophy, X-LinkedDatasets as TopicFemaleHumansMaleMiddle AgedMuscular Atrophy, SpinalMuscular Disorders, AtrophicTreatment Outcome

Identifiers

PMID39591556
PMCPMC11666247

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