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Responsiveness and Minimal Clinically Important Difference of the Motor Function Measure in collagen VI-related dystrophies (COL6-RD) and laminin alpha2-related muscular dystrophy (LAMA2-RD)
Le Goff L, Meilleur KG, Norato G, et al.
Archives of physical medicine and rehabilitation, 2020
Effects of nusinersen after one year of treatment in 123 children with SMA type 1 or 2: a French real-life observational study
Audic F, De la Banda MGG, Bernoux D, et al.
Orphanet journal of rare diseases, 2020, 15, 1, p 148
Palliative Care in SMA Type 1: A Prospective Multicenter French Study Based on Parents' Reports
Hully M, Barnerias C, Chabalier D, et al.
Frontiers in pediatrics, 2020, 8, 4
Validity and Reliability of the 32-Item Motor Function Measure in 2- to 5-Year-Olds with Neuromuscular Disorders and 2- to 25-Year-Olds with Spinal Muscular Atrophy
Trundell D, Le Scouiller S, Gorni K, et al.
Neurology and therapy, 2020
Assessment of the validity and reliability of the 32-item Motor Function Measure in individuals with Type 2 or non-ambulant Type 3 spinal muscular atrophy
Trundell D, Trundell D, Le Scouiller S, et al.
PLoS ONE, 2020, 15, 9
SMA - Des échelles d’évaluation motrice pour le public francophone : French adaptation of motor function scales for patients with spinal muscular atrophy
Pons C, Barrière A, Bertrand G, et al.
Les Cahiers de Myologie, 2019, 35, HS2, p 24
Cross-cultural Adaptation and Multi-centric Validation of the Motor Function Measure Chinese Version (MFM-32-CN) for Patients with Neuromuscular Diseases
Huang M, Cao J, Sun J, et al.
Developmental neurorehabilitation, 2019
Motor function performance in individuals with RYR1-related myopathies
Witherspoon JW, Vuillerot C, Vasavada RP, et al.
Muscle & Nerve, 2019, 60, 1, p 80
X-linked myotubular myopathy: A prospective international natural history study
Annoussamy M, Lilien C, Gidaro T, et al.
Neurology, 2019, 92, 16
Measurements of Motor Function and Other Clinical Outcome Parameters in Ambulant Children with Duchenne Muscular Dystrophy
Nagy S, Schmidt S, Hafner P, et al.
Journal of visualized experiments : JoVE, 2019, 143
Longitudinal changes in clinical outcome measures in COL6-related dystrophies and LAMA2-related dystrophies
Jain MS, Meilleur K, Kim E, et al.
Neurology, 2019, 93, 21
Nusinersen in patients older than 7 months with spinal muscular atrophy type 1: A cohort study.
Aragon-Gawinska K, Seferian AM, Daron A, et al.
Neurology, 2018, e1312
Prospective and longitudinal natural history study of patients with Type 2 and 3 spinal muscular atrophy: Baseline data NatHis-SMA study.
Chabanon A, Seferian AM, Daron A, et al.
PLoS ONE, 2018, e0201004
Mathematical Disease Progression Modeling in Type 2/3 Spinal Muscular Atrophy
Jacqmin P, Jacqmin P, Gieschke R, et al.
Muscle & Nerve, 2018
Congenital titinopathy: Comprehensive characterisation and pathogenic insights
Oates EC, Jones KJ, Donkervoort S, et al.
Annals of neurology, 2018
Is Going Beyond Rasch Analysis Necessary to Assess the Construct Validity of a Motor Function Scale?
Guillot T, Roche S, Rippert P, et al.
Archives of physical medicine and rehabilitation, 2018
Corticosteroids in Duchenne muscular dystrophy: impact on the motor function measure sensitivity to change and implications for clinical trials
Schreiber A, Brochard S, Rippert P, et al.
Developmental medicine and child neurology, 2018, 60, 2, p 185
Minimal clinically important difference for the Motor Function Measure in patients with congenital muscular dystrophy and congenital myopathy : Abstract P326 extrait du colloque 23rd WMS Congress 2-6 october 2018 Argentina
Le Goff L, Fink M, Norato G, et al.
Neuromuscular disorders : NMD, 2018, 28
La juste mesure pour un meilleur accompagnement : Editorial
Vuillerot C, Poirot I
Les Cahiers de Myologie, 2017, 33, HS 1, p 5
Electrical impedance myography in individuals with collagen 6 and laminin alpha-2 congenital muscular dystrophy: a cross-sectional and 2-year analysis
Nichols C, Jain MS, Meilleur KG, et al.
Muscle & Nerve, 2017, 57, 1, p 54
First experience of Nusinersen early access program in patients with spinal muscular atrophy type 1
Gargaun E, Aragon-Gawinska K, Seferian A, et al.
Neuromuscular disorders : NMD, 2017, 27, s2, S211
Safety, tolerability and clinical efficacy of nusinersen in SMA type 1 older than 7 months: a prospective study
Aragon-Gawinska K, Gargaun E, Seferian A, et al.
Neuromuscular disorders : NMD, 2017, 27, s2, s211
Safety and efficacy of olesoxime in patients with type 2 or non-ambulatory type 3 spinal muscular atrophy: a randomised, double-blind, placebo-controlled phase 2 trial
Bertini E, Dessaud E, Mercuri E, et al.
The Lancet. Neurology, 2017, 16, 7, p 513
2005-2015: Ten years clinical experience in treating DMD patients by corticosteroids in Lyon
Fontaine Carbonnel S, Rippert P, Poirot I, et al.
Annals of physical and rehabilitation medicine, 2016, 59S, p e82
Innovative method for motor functions evaluation in SMA type 2 and 3 patients
Vuillerot C, Vincent-Genod D, Thomann G, et al.
Annals of physical and rehabilitation medicine, 2016, 59S, p e84
