Innovative methods to assess upper limb strength and function in non-ambulant Duchenne patients

Servais, Laurent; Deconinck, Nicolas; Moraux, A.; Benali, M.; Canal, A.; Parys, F. Van; Vereecke, W.; Wittevrongel, S.; Mayer, M.; Desguerre, Isabelle; Maincent, Kim; Thémar-Noël, Christine; Quijano‐Roy, Susana; Serari, N; Voït, Thomas; Hogrel, Jean‐Yves

doi:10.1016/j.nmd.2012.10.022

Cited by 83 publications

(120 citation statements)

References 21 publications

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“…As for the MFM, the total score and the D1 showed a moderate change over 4 years. This study did not include tests that were subsequently generalized for the evaluation of muscular dystrophies such as the 6‐min walk test or the myoset 38, 39. They would probably be very useful tools in the case of LGMD2A, especially considering the modification of walking perimeter seen in the patients and the very slow progression of the disease.…”

Section: Discussionmentioning

confidence: 99%

Natural history of LGMD2A for delineating outcome measures in clinical trials

Richard

Hogrel

Stockholm

et al. 2016

Ann Clin Transl Neurol

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ObjectiveLimb‐girdle muscular dystophy 2A (LGMD2A, OMIM) is a slowly progressive myopathy caused by the deficiency in calpain 3, a calcium‐dependent cysteine protease of the skeletal muscle.MethodsIn this study, we carried out an observational study of clinical manifestations and disease progression in genetically confirmed LGMD2A patients for up to 4 years. A total of 85 patients, aged 14–65 years, were recruited in three centers located in metropolitan France, the Basque country, and the Reunion Island. They were followed up every 6 months for 2 years and a subgroup was assessed annually thereafter for two more years. Data collected for all patients included clinical history, blood parameters, muscle strength assessed by manual muscle testing (MMT) and quantitative muscle testing, functional scores, and pulmonary and cardiac functions. In addition, CT scans of the lower limbs were performed in a subgroup of patients.ResultsOur study confirms the clinical description of a slowly progressive disorder with onset in the first or second decade of life with some degree of variability related to gender and mutation type. The null mutations lead to a more severe phenotype while compound heterozygote patients are the least affected. Muscle weakness is remarkably symmetrical and predominant in the axial muscles of the trunk and proximal muscles of the lower limb. There was a high correlation between the weakness at individual muscle level as assessed by MMT and the loss of density in CT scan analysis.InterpretationAll the generated data will help to determine the endpoints for further clinical studies.

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Section: Discussionmentioning

confidence: 99%

Natural history of LGMD2A for delineating outcome measures in clinical trials

Richard

Hogrel

Stockholm

et al. 2016

Ann Clin Transl Neurol

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“…Основными являются методы функциональной оценки, для которых разработано большое число из-мерительных устройств и протоколов, причем многие из них оптимизированы для измерения отдельных движений, тогда как другие направлены на выявление глобальных изменений в активности пациента [14][15][16][17][18][19][20][21][22][23][24][25]. Методы функциональной оценки относятся к акти-метрии, новой быстро развивающейся дисциплине, и часто становятся единственным способом оценки состояния пациента в привычной для него среде через определенные периоды времени.…”

Section: лекции и обзорыunclassified

Skeletal Muscle Quantitative Nuclear Magnetic Resonance Imaging and Spectroscopy as an Outcome Measure for Clinical Trials (Part I)

Carlier¹,

Marty²,

Scheidegger³

et al. 2016

Neuromuscular Diseases

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“…, Mariana C. Voos 1 , Fátima A. Caromano 1 In the last years, the importance of accurate, reliable and sensitive outcome measures in patients with Duchenne muscular dystrophy (DMD) has been outlined 1,2,3 . Motor function is, currently, the main outcome measure (e.g.…”

mentioning

confidence: 99%

“…Motor Function Measure, MFM 2,4,5 , six-minute walking test 6 and North Star ambulatory assessment 7,8 ). However, motor function scores can be complemented by clinical measures, such as muscle strength, evaluated by the Medical Research Council scale (MRC) 9,10,11,12,13 , to provide more detailed monitoring of disease progression 2,3 . In DMD, muscle strength is assessed to describe weakness topography and disease progression rate.…”

mentioning

confidence: 99%

Relationship between muscle strength and motor function in Duchenne muscular dystrophy

Nunes

Hukuda

Fávero

et al. 2016

Arq. Neuro-Psiquiatr.

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Measuring muscle strength and motor function is part of Duchenne muscular dystrophy (DMD) assessment. However, the relationship between these variables is controversial. Objective To investigate the relationship between muscle strength and motor function and between these variables and age. Method Muscle strength was measured by Medical Research Council (MRC) scale and motor function, by Motor Function Measure (MFM), in 40 non-ambulatory patients. Spearman tests investigated the relationships between muscle strength, motor function and age. Results Total MRC and MFM scores were strongly related to each other (r = 0.94; p < 0.001), but not to age (r = -0.19, r = -0.31, respectively; p > 0.05). Strong and moderate relationships between partial muscle strength and motor function scores were found. Higher correlation coefficients were found between total scores and Dimensions 2 (axial/ proximal control) and 3 (distal control) of MFM. Conclusion Muscle strength and motor function are strongly correlated and seem to decrease proportionally in DMD.

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Innovative methods to assess upper limb strength and function in non-ambulant Duchenne patients

Cited by 83 publications

References 21 publications

Natural history of LGMD2A for delineating outcome measures in clinical trials

Natural history of LGMD2A for delineating outcome measures in clinical trials

Skeletal Muscle Quantitative Nuclear Magnetic Resonance Imaging and Spectroscopy as an Outcome Measure for Clinical Trials (Part I)

Relationship between muscle strength and motor function in Duchenne muscular dystrophy

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