Idebenone for the treatment of Duchenne muscular dystrophy (Protocol)
Geng J, Dong J, Jiang K, Shen L, Wu T, Ni H, Shi LL, Wang G, Wu H.
The Cochrane Library 2011.
Description of a protocol aiming at assessing efficacy of ibedenone on muscle strength, muscle function, lung function, cardiac function and quality of life for patients with DMD. The MFM is use as primary outcome to assess motor function of patients after 12 months of treatment.
Keywords: Duchenne Muscular Dystrophy, Idebenone, Treatment efficacy, Protocol, Clinical trial
Articles dans les revues scientifiques
Hyperleptinemia in children with autosomal recessive spinal muscular atrophy type I-III
Kölbel H, Hauffa BP, Wudy SA, Bouikidis A, Della Marina A, Schara U PLoS One. 2017. 9;12(3):e0173144. Study of prevalence of hyperleptinemia in a spinal muscular atrophy (SMA) population.Hyperleptinemia is highly prevalent in underweight children with SMA and is...
Electrical impedance myography (EIM) in individuals with COL6 and LAMA2 congenital muscular dystrophy: a cross-sectional and two-year analysis
Nichols C, Jain MS, Meilleur KG, Wu T, Collins J, Waite MR, Dastgir J, Salman A, Donkervoort S, Duong T, Keller K, Leach ME, Lott DJ, McGuire MN, Nelson L, Rutkowski A, Vuillerot C, Bönnemann CG, Lehky TJ. Muscle Nerve. 2018. 57(1):54-60. The main objective of the...
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...