Modalités de l’utilisation de l’alpha-glucosidase recombinante humaine (Myosine TM) et du suivi des patients adultes
Annane D, Caillaud C, Laforêt P, Maire I, Nicolino M, Orlikovski D.
Recommandations du Comité d’Evaluation du traitement de la maladie de Pompe 2008.
In adult Pompe disease, it is recommended to assess patients by MFM initially and every 6 months or annually by the reference center for the patient in the monitoring of the Myosine protocol. (in French).
Keywords : Pompe disease, Myosyme, recommendations, protocol, clinical trials
Articles dans les revues scientifiques
Molecular therapeutic strategies for spinal muscular atrophies: current and future clinical trials
Zanetta C, Nizzardo M, Simone C, Monguzzi E, Bresolin N, Comi GP, Corti S. Clin Ther. 2014. 1;36(1):128-40. This article provides a general overview of the main aspects that need to be taken into account to design a more efficient clinical trial and to summarize the...
Relationship between muscle strength and motor function in Duchenne muscular dystrophy
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Influence of a two-year steroid treatment on body composition as measured by Dual X-Ray Absorptiometry in boys with Duchenne Muscular Dystrophy
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The applicability of four clinical methods to evaluate arm and hand function in all stages of spinal muscular atrophy type II.
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Diaphragmatic dysfunction in Collagen VI myopathies
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Hammersmith Functional Motor Scale and Motor Function Measure-20 in non ambulant SMA patients
E. Mazzone, R. De Sanctis, L. Fanelli, F. Bianco, M. Main, M. van den Hauwe, M. Ash, R. de Vries, J. Fagoaga Mata, K. Schaefer, A. D’Amico, G. Colia, C. Palermo, M. Scoto, A. Mayhew, M. Eagle, L. Servais, M. Vigo, A. Febrer, R. Korinthenberg, M. Jeukens M. de Viesser,...