A new score combining compound muscle action potential (CMAP) amplitudes and motor score is predictive of motor outcome after AVXS-101 (Onasemnogene Abeparvovec) SMA therapy
暂无分享,去创建一个
Y. Péréon | P. Cintas | C. Barnérias | I. Desguerre | C. Boulay | R. Barrois | C. Vuillerot | J. Davion | F. Audic | V. Manel | U. Walther-Louvier | C. Gitiaux | C. Espil-Taris | C. Cances | S. Roy | E. Deladrière | J. Piarroux | B. Tervil | V. Leloup-Germa
[1] S. Mercier,et al. Fatal thrombotic microangiopathy case following adeno-associated viral SMN gene therapy , 2022, Blood advances.
[2] F. Heinen,et al. Quantitative Motion Measurements Based on Markerless 3D Full-Body Tracking in Children with SMA Highly Correlate with Standardized Motor Assessments. , 2021, Journal of neuromuscular diseases.
[3] B. Chabrol,et al. Motor unit number index: A potential electrophysiological biomarker for pediatric spinal muscular atrophy , 2021, Muscle & nerve.
[4] R. Finkel,et al. Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, phase 3 trial , 2021, The Lancet Neurology.
[5] D. Kariyawasam,et al. Motor unit changes in children with symptomatic spinal muscular atrophy treated with nusinersen , 2020, Journal of Neurology, Neurosurgery, and Psychiatry.
[6] A. Ziegler,et al. European ad-hoc consensus statement on gene replacement therapy for spinal muscular atrophy , 2020, European Journal of Paediatric Neurology.
[7] M. Ryan,et al. Biomarkers and the Development of a Personalized Medicine Approach in Spinal Muscular Atrophy , 2019, Front. Neurol..
[8] Markus Weber,et al. Motor Unit Number Index (MUNIX) of hand muscles is a disease biomarker for adult spinal muscular atrophy , 2019, Clinical Neurophysiology.
[9] J. Mendell,et al. AVXS-101 (Onasemnogene Abeparvovec) for SMA1: Comparative Study with a Prospective Natural History Cohort. , 2019, Journal of neuromuscular diseases.
[10] Craig McDonald,et al. Natural history of infantile‐onset spinal muscular atrophy , 2017, Annals of neurology.
[11] Brian K. Kaspar,et al. Single‐Dose Gene‐Replacement Therapy for Spinal Muscular Atrophy , 2017, The New England journal of medicine.
[12] R. J. Ramamurthi,et al. Nusinersen versus Sham Control in Infantile‐Onset Spinal Muscular Atrophy , 2017, The New England journal of medicine.
[13] D. Vissière,et al. P.429 Innovative home activity monitoring in non-ambulant patients with spinal muscular atrophy: a multicenter observational trial , 2017, Neuromuscular Disorders.
[14] N. Vayatis,et al. An Automated Recording Method in Clinical Consultation to Rate the Limp in Lower Limb Osteoarthritis , 2016, PloS one.
[15] J. Kissel,et al. Spinal Muscular Atrophy. , 2015, Neurologic clinics.
[16] W. Chung,et al. Observational study of spinal muscular atrophy type I and implications for clinical trials , 2014, Neurology.
[17] W. Chung,et al. The motor neuron response to SMN1 deficiency in spinal muscular atrophy , 2014, Muscle & nerve.
[18] Xiaobai Li,et al. Electrophysiological biomarkers in spinal muscular atrophy: proof of concept , 2013, Annals of clinical and translational neurology.
[19] M. Schroth,et al. Compound muscle action potential and motor function in children with spinal muscular atrophy , 2010, Muscle & nerve.
[20] Mark B Bromberg,et al. Natural history of denervation in SMA: Relation to age, SMN2 copy number, and function , 2005, Annals of neurology.