Free Access
Issue
Med Sci (Paris)
Volume 35, Novembre 2019
Les Cahiers de Myologie
Page(s) 24 - 28
Section Prise en charge
DOI https://doi.org/10.1051/medsci/2019189
Published online 20 December 2019
  1. Finkel RS, Mercuri E, Darras BT, et al., Nusinersen versus sham control in infantile-onset spinal muscular atrophy. N Engl J Med . 2017;377:1723–32. [Google Scholar]
  2. Mercuri E, Darras BT, Chiriboga CA, et al., Nusinersen versus sham control in later-onset spinal muscular atrophy. N Engl J Med . 2018;378:625–35. [Google Scholar]
  3. Montes J, Dunaway Young S, Mazzone ES, et al., Nusinersen improves walking distance and reduces fatigue in later-onset SMA. Muscle Nerve . 2019; Jul :12. doi: 10.1002/mus.26633. [Google Scholar]
  4. Haataja L, Mercuri E, Regev R, et al., Optimality score for the neurologic examination of the infant at 12 and 18 months of age. J Pediatr . 1999;135:153–61. [CrossRef] [PubMed] [Google Scholar]
  5. Glanzman AM, Mazzone ES, Young SD, et al., Evaluator training and reliability for SMA global nusinersen trials1. J Neuromuscul Dis . 2018;5:159–66. [CrossRef] [PubMed] [Google Scholar]
  6. O’Hagen JM, Glanzman AM, McDermott MP, et al., An expanded version of the hammersmith functional motor scale for SMA II and III patients. Neuromuscul Disord . 2007;17:693–7. [CrossRef] [PubMed] [Google Scholar]
  7. Mazzone ES, Mayhew A, Montes J, et al., Revised upper limb module for spinal muscular atrophy: development of a new module. Muscle Nerve . 2017;55:869–74. [Google Scholar]
  8. McDonald CM, Henricson EK, Han JJ, et al., The 6-minute walk test as a new outcome measure in Duchenne muscular dystrophy. Muscle Nerve . 2010;41:500–10. [Google Scholar]
  9. Bérard C, Payan C, Hodgkinson I, Fermanian J, and MFM Collaborative Study Group. A motor function measure for neuromuscular diseases. Construction and validation study. Neuromuscul Disord . 2005;15:463–70. [CrossRef] [PubMed] [Google Scholar]
  10. Bishop KM, Montes J, Finkel RS, Motor milestone assessment of infants with spinal muscular atrophy using the hammersmith infant neurological exam. Part 2. Experience from a nusinersen clinical study. Muscle Nerve . 2018;57:142–6. [Google Scholar]
  11. Dunaway Young S, Montes J, Kramer SS, et al., Six-minute walk test is reliable and valid in spinal muscular atrophy. Muscle Nerve . 2016;54:836–42. [Google Scholar]
  12. Glanzman AM, Mazzone E, Main M, et al., The Children’s hospital of Philadelphia infant test of neuromuscular disorders (CHOP INTEND): test development and reliability. Neuromuscul Disord . 2010;20:155–61. [CrossRef] [PubMed] [Google Scholar]
  13. Vuillerot C, Payan C, Iwaz J, Ecochard R, Bérard Cand MFM Spinal Muscular Atrophy Study Group, Responsiveness of the motor function measure in patients with spinal muscular atrophy. Arch Phys Med Rehabil . 2013;94:1555–61. [CrossRef] [PubMed] [Google Scholar]
  14. Darras BT, Chiriboga CA, Iannaccone ST, et al., Nusinersen in later-onset spinal muscular atrophy: long-term results from the phase 1/2 studies. Neurology . 2019;92:e2492–506. [Google Scholar]
  15. Pane M, Palermo C, Messina S, et al., Nusinersen in type 1 SMA infants, children and young adults: preliminary results on motor function. Neuromuscul Disord . 2018;28:582–5. [CrossRef] [PubMed] [Google Scholar]
  16. Pechmann A, Langer T, Schorling D, et al., Evaluation of children with SMA type 1 under treatment with nusinersen within the expanded access program in Germany. J Neuromuscul Dis . 2018;5:135–43. [CrossRef] [PubMed] [Google Scholar]
  17. Pane M, Coratti G, Sansone VA, et al., Nusinersen in type 1 spinal muscular atrophy: twelve-month real-world data. Ann Neurol . 2019;86:443–51. [CrossRef] [PubMed] [Google Scholar]
  18. Mazzone E, Bianco F, Martinelli D, et al., Assessing upper limb function in nonambulant SMA patients: development of a new module. Neuromuscul Disord . 2011;21:406–12. [CrossRef] [PubMed] [Google Scholar]
  19. Wild D, Grove A, Martin M, Eremenco S, McElroy S, Verjee-Lorenz A, Erikson P, and ISPOR Task Force for Translation and Cultural Adaptation, Principles of good practice for the translation and cultural adaptation process for patient-reported outcomes (PRO) measures: report of the ISPOR task force for translation and cultural adaptation. Value Health . 2005;8:94–104. [CrossRef] [PubMed] [Google Scholar]

Current usage metrics show cumulative count of Article Views (full-text article views including HTML views, PDF and ePub downloads, according to the available data) and Abstracts Views on Vision4Press platform.

Data correspond to usage on the plateform after 2015. The current usage metrics is available 48-96 hours after online publication and is updated daily on week days.

Initial download of the metrics may take a while.