Assessing Dysferlinopathy Patients Over Three Years With a New Motor Scale

Author:

Jacobs Marni B.12ORCID,James Meredith K.3ORCID,Lowes Linda P.4ORCID,Alfano Lindsay N.4ORCID,Eagle Michelle3,Muni Lofra Robert3,Moore Ursula3,Feng Jia1,Rufibach Laura E.5,Rose Kristy6,Duong Tina78,Bello Luca9ORCID,Pedrosa‐Hernández Irene10,Holsten Scott11,Sakamoto Chikako12,Canal Aurélie13,Sanchez‐Aguilera Práxedes Nieves14,Thiele Simone15,Siener Catherine16,Vandevelde Bruno17,DeWolf Brittney7,Maron Elke18,Guglieri Michela3,Hogrel Jean‐Yves13ORCID,Blamire Andrew M.19ORCID,Carlier Pierre G.20,Spuler Simone21,Day John W.22,Jones Kristi J.6,Bharucha‐Goebel Diana X.2324,Salort‐Campana Emmanuelle17,Pestronk Alan16ORCID,Walter Maggie C.15,Paradas Carmen25,Stojkovic Tanya13,Mori‐Yoshimura Madoka26,Bravver Elena11,Díaz‐Manera Jordi2728,Pegoraro Elena9,Mendell Jerry R.4,Mayhew Anna G.3,Straub Volker3ORCID,

Affiliation:

1. Center for Translational Science, Division of Biostatistics and Study Methodology Children's National Health System Washington DC

2. Pediatrics, Epidemiology, and Biostatistics George Washington University Washington DC

3. The John Walton Muscular Dystrophy Research Centre, Translational and Clinical Research Institute Newcastle University and Newcastle Hospitals NHS Foundation Trust, Central Parkway Newcastle upon Tyne UK

4. The Abigail Wexner Research Institute at Nationwide Children's Hospital Columbus OH

5. The Jain Foundation Seattle WA

6. The Children's Hospital at Westmead The University of Sydney Sydney Australia

7. Cooperative International Neuromuscular Research Group (CINRG) Children's National Health System Washington DC

8. Lucile Salter Packard Children's Hospital at Stanford Palo Alto CA

9. Department of Neuroscience University of Padova Padova Italy

10. Physical Medicine and Rehabilitation Hospital de la Santa Creu i Sant Pau Barcelona Spain

11. Neuroscience Institute, Carolinas Neuromuscular/ALS‐MDA Center Carolinas HealthCare System Charlotte NC

12. Department of Physical Rehabilitation, National Center Hospital National Center of Neurology and Psychiatry Tokyo Japan

13. Institut de Myologie, AP‐HP, GH Pitié‐Salpêtrière Paris France

14. Neurorehabilitation Unit Rehabilitation Hospital Universitario Virgen del Rocío Sevilla Spain

15. Friedrich‐Baur‐Institute, Department of Neurology Ludwig‐Maximilians University of Munich Munich Germany

16. Department of Neurology Washington University School of Medicine St. Louis MO

17. Service des Maladies Neuromusculaire et de la SLA Hôpital de La Timone Marseille France

18. ELAN‐PHYSIO, Praxis für Physiotherapie Maron Berlin Germany

19. Magnetic Resonance Centre, Institute for Cellular Medicine Newcastle University Newcastle upon Tyne UK

20. AIM & CEA NMR Laboratory, Institute of Myology Pitié‐Salpêtrière University Hospital Paris France

21. Charite Muscle Research Unit, Experimental and Clinical Research Center, a joint cooperation of the Charité Medical Faculty and the Max Delbrück Center for Molecular Medicine Berlin Germany

22. Department of Neurology and Neurological Sciences Stanford University School of Medicine Stanford CA

23. Department of Neurology Children's National Health System Washington DC

24. National Institutes of Health (NINDS) Bethesda MD

25. Neuromuscular Unit, Department of Neurology Hospital U. Virgen del Rocío/Instituto de Biomedicina de Sevilla Sevilla Spain

26. Department of Neurology National Center Hospital, National Center of Neurology and Psychiatry Tokyo Japan

27. Centro de Investigación Biomédica en Red en Enfermedades Raras (CIBERER) Barcelona Spain

28. Neuromuscular Disorders Unit, Neurology Department Hospital de la Santa Creu i Sant Pau Barcelona Spain

Publisher

Wiley

Subject

Neurology (clinical),Neurology

Reference31 articles.

1. One gene, one or many diseases?: Simplifying dysferlinopathy

2. Dysferlin mutations in LGMD2B, Miyoshi myopathy, and atypical dysferlinopathies

3. The limb‐girdle muscular dystrophies and the dystrophinopathies;Iyadurai SJ;Continuum,2016

4. The limb‐girdle muscular dystrophies;Wicklund MP;Continuum,2019

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