- •37 adults with SMA 2–3 treated with nusinersen, followed for up to 30 months of treatment.
- •Median MRC score increased from baseline to visits at 6 and 14 months, not afterwards.
- •The median rate of MRC score increase was 1.85 points per year.
- •Revised hammersmith scale median score increased only from baseline to 6 months.
- •Revised hammersmith scale's calculated annual rate of change was 0 points.
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- Spinal muscular atrophy.The Lancet, 2008 (Vol. 371)
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- Hammersmith functional motor scale and motor function measure-20 in non ambulant SMA patients.Neuromuscul Disord. 2014; 24
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- A natural history study of late onset spinal muscular atrophy types 3b and 4.J Neurol. 2008; 255
- Advances in therapy for spinal muscular atrophy: promises and challenges.Nature Rev Neurol. 2018;
- Nusinersen versus sham control in infantile-onset spinal muscular atrophy.N Engl J Med. 2017; 377
- Nusinersen versus sham control in later-onset spinal muscular atrophy.N Engl J Med. 2018; 378
- Evidence in focus: nusinersen use in spinal muscular atrophy report of the guideline development, dissemination, and implementation subcommittee of the american academy of neurology.Neurology. 2018; 91
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- Nusinersen treatment significantly improves hand grip strength, hand motor function and MRC sum scores in adult patients with spinal muscular atrophy types 3 and 4.J Neurol [Internet]. 2020; 268 (Available from:): 923-935https://doi.org/10.1007/s00415-020-10223-9
- Nusinersen safety and effects on motor function in adult spinal muscular atrophy type 2 and 3.J Neurol Neurosurg Psychiatry. 2020; 91: 1166-1174
- Nusinersen in adults with 5q spinal muscular atrophy: a non-interventional, multicentre, observational cohort study.Lancet Neurol [Internet]. 2020; 19 (Available from:): 317-325https://doi.org/10.1016/S1474-4422(20)30037-5
- Nusinersen for older patients with spinal muscular atrophy: a real-world clinical setting experience.Muscle Nerve. 2020; 61
- SMArtCARE - A platform to collect real-life outcome data of patients with spinal muscular atrophy.Orphanet J Rare Dis. 2019; 14
- Consensus statement for standard of care in spinal muscular atrophy.J Child Neurol. 2007; 22
Finkel R.S., Sejersen T., Mercuri E., Bertini E., Chen K., Crawford T.O., et al. 218th ENMC International Workshop:: Revisiting the consensus on standards of care in SMA Naarden, The Netherlands, 19–21 February 2016. In: neuromuscular Disorders. 2017.
- Muscle strength and motor function in adolescents and adults with spinal muscular atrophy.Neurology. 2020; 95: e1988-e1998
- Revised Hammersmith scale for spinal muscular atrophy.Neuromuscul Disord. 2015; 25
- Revised Hammersmith Scale for spinal muscular atrophy: a SMA specific clinical outcome assessment tool.PLoS ONE. 2017; 12
- The ALSFRS-R: a revised ALS functional rating scale that incorporates assessments of respiratory function. BDNF ALS Study Group (Phase III).J Neurol Sci. 1999; 169
- Nusinersen treatment of older children and adults with spinal muscular atrophy.Neuromuscul Disord [Internet]. 2020; (xxxx). Available from:https://doi.org/10.1016/j.nmd.2020.12.006
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FDA. Nusinersen BLA. 2015; Available from: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2015/207958Orig1s000ChemR.pdf