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
Cross-sectional retrospective study of muscle function in patients with glycogen storage disease type III
Decostre V, Laforêt P, Nadaj-Pakleza A, De Antonio M, Leveugle S, Ollivier G, Canal A, Kachetel K, Petit F, Eymard B, Behin A, Wahbi K, Labrune P, Hogrel JY Neuromuscul Disord. 2016. 26(9):584-92. This study aimed to identify promising muscle function measures for...
Facioscapulohumeral dystrophy in children: design of a prospective, observational study on natural history, predictors and clinical impact (iFocus FSHD)
Goselink RJ, Schreuder TH, Mul K, Voermans NC, Pelsma M, de Groot IJ, van Alfen N, Franck B, Theelen T, Lemmers RJ, Mah JK, van der Maarel SM, van Engelen BG, Erasmus CE. BMC Neurol. 2016. 17;16:138. Protocol of a population-based prospective cohort study on...
Muscle MRI Findings in Childhood/Adult Onset Pompe Disease Correlate with Muscle Function
Figueroa-Bonaparte S, Segovia S, Llauger J, Belmonte I, Pedrosa I, Alejaldre A, Mayos M, Suárez-Cuartín G, Gallardo E, Illa I, Díaz-Manera J; Spanish Pompe Study Group. PLoS One. 2016. 6;11(10):e0163493. The main objective of the study was to assess the use of muscle...
The 6-minute walk test, motor function measure and quantitative thigh muscle MRI in Becker muscular dystrophy: A cross-sectional study
Fischer D, Hafner P, Rubino D, Schmid M, Neuhaus C, Jung H, Bieri O, Haas T, Gloor M, Fischmann A, Bonati U. Neuromuscul Disord. 2016. 26(7):414-22. This study investigated the relation between validated functional scores, such as MFM, timed function tests, such as...
Therapy Taping Method: Therapeutic approach in two children with Duchenne Muscular Dystrophy
Iwabe-Marchese C, Morini NJr. Br J Med Med Res. 2016. 15(3): 1-7. The aim of the study is to assess the effect of elastic bandage through the Therapy Taping Method in two children with DMD. The Motor Function Measure-20 scale (MFM-20) was used as an outcome...
Natural history of LGMD2A for delineating outcome measures in clinical trials
Richard I, Hogrel JY, Stockholm D, Payan CA, Fougerousse F; Calpainopathy Study Group, Eymard B, Mignard C, Lopez de Munain A, Fardeau M, Urtizberea JA. Ann Clin Transl Neurol. 2016. 4;3(4):248-65. Observational study of clinical manifestations and disease progression...