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
Short-TERM Neuromuscular Electrical Stimulation Training of the Tibialis Anterior Did Not Improve Strength and Motor Function in Facioscapulohumeral Muscular Dystrophy Patients
Doix AM, Roeleveld K, Garcia J, Lahaut P, Tanant V, Fournier-Mehouas M, Desnuelle C, Colson SS, Sacconi S Am J Phys Med Rehabil. 2017. 96(4):e56-e63. Prospective study including 10 patients with facioscapulohumeral muscular dystrophy type 1 (FSHD1) and 10 healthy...
Upper extremity outcome measures for collagen VI-related myopathy and LAMA2-related muscular dystrophy
Bendixen RM, Butrum J, Jain MS, Parks R, Hodsdon B, Nichols C, Hsia M, Nelson L, Keller KC, McGuire M, Elliott JS, Linton MM, Arveson IC, Tounkara F, Vasavada R, Harnett E, Punjabi M, Donkervoort S, Dastgir J, Leach ME, Rutkowski A, Waite M, Collins J, Bönnemann CG,...
Reliability of home-based, motor function measure in hereditary neuromuscular diseases
Ruiz-Cortes X, Ortiz-Corredor F, Mendoza-Pulido C J Int Med Res. 2017. 45(1):261-271. The goal of the study was to compare the reliability of the MFM scale in the assessment of patients when administered at home and clinic. Results demonstrated excellent inter-rater...
Longitudinal Functional and NMR Assessment of Upper Limbs in Duchenne Muscular Dystrophy
Hogrel JY, Wary C, Moraux A, Azzabou N, Decostre V, Ollivier G, Canal A, Lilien C, Ledoux I, Annoussamy M, Reguiba N, Gidaro T, Le Moing AG, Cardas R, Voit T, Carlier PG, Servais L. Neurology. 2016. 15;86(11):1022-30. Twenty-five 53-skippable patients with DMD were...
Treatment with L-citrulline in patients with post-polio syndrome: study protocol for a single-center, randomised, placebo-controlled, double-blind trial
Schmidt S, Gocheva V, Zumbrunn T, Rubino-Nacht D, Bonati U, Fischer D, Hafner P Trials. 2017. 9;18(1):116. Protocol of a randomized, double-blind, placebo-controlled trial aiming to demonstrate determine if treatment with L-citrulline shows a positive effect on...
Postrehabilitation Functional Improvements in Patients With Inflammatory Myopathies: The Results of a Randomized Controlled Trial
Tiffreau V, Rannou F, Kopciuch F, Hachulla E, Mouthon L, Thoumie P, Sibilia J, Drumez E, Thevenon A Arch Phys Med Rehabil. 2017. 98(2):227-234. Multicenter randomized controlled trial, aiming to evaluate the medium-term functional effect and the effect on quality of...