Assessing Dysferlinopathy Patients Over Three Years With a New Motor Scale
Annals of Neurology2021Vol. 89(5), pp. 967–978
Citations Over TimeTop 15% of 2021 papers
Marni Jacobs, M. James, Linda Lowes, Lindsay N. Alfano, Michelle Eagle, R. Muni Lofra, U. Moore, Jia Feng, Laura Rufibach, Kristy Rose, Tina Duong, Luca Bello, Irene Pedrosa‐Hernández, Scott Holsten, Chikako Sakamoto, Aurélie Canal, Nieves Sanchez‐Aguilera Práxedes, Simone Thiele, Catherine Siener, Bruno Vandevelde, Brittney DeWolf, Elke Maron, Michela Guglieri, Jean‐Yves Hogrel, Andrew M. Blamire, Pierre G. Carlier, Simone Spuler, John Day, Kristi Jones, Diana Bharucha‐Goebel, Emmanuelle Salort‐Campana, Alan Pestronk, Maggie C. Walter, Carmen Paradas, Tanya Stojkovic, Madoka Mori‐Yoshimura, Elena Bravver, Jordi Díaz‐Manera, Elena Pegoraro, Jerry R. Mendell, Anna Mayhew, Volker Straub
Abstract
The NSAD is the first validated limb girdle specific scale of motor performance, suitable for use in clinical practice and clinical trials. Longitudinal analysis showed it may be possible to identify patient factors associated with greater functional decline both across the disease course and in the short-term for clinical trial preparation. Through further work and validation in this cohort, we anticipate that a disease model incorporating functional performance will allow for more accurate prognosis for patients with dysferlinopathy. ANN NEUROL 2021;89:967-978.
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