- •The aim was to establish 24-month changes in type II and III SMA patients.
- •107 patients (54 type II and 53 type III) were grouped by type, function and age.
- •23% of patients decreased more than 2 points and 7% improving by >2 points.
- •Significant changes were observed after 24 but not after 12 months.
- •Our results suggest a progressive decline in upper limb function.
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- Spinal muscular atrophy.Orphanet J Rare Dis. 2011; 6: 71
- Chaos in the classification of SMA: a possible resolution.Neuromuscul Disord. 1995; 5: 3-5
- Assessing upper limb function in nonambulant SMA patients: development of a new module.Neuromuscul Disord. 2011; 21: 406-412
- Revised upper limb module for spinal muscular atrophy: development of a new module.Muscle Nerve. 2016;
- Reliability of hand-held dynamometry in spinal muscular atrophy.Muscle Nerve. 2002; 26: 64-70
- Revised upper limb module for spinal muscular atrophy: 12 month changes.Muscle Nerve. 2019; 59: 426-430
- Upper limb module in non-ambulant patients with spinal muscular atrophy: 12 month changes.Neuromuscul Disord. 2015; 25: 212-215
- Evaluator training and reliability for SMA global nusinersen trials1.J Neuromuscul Dis. 2018; 5: 159-166
- Hausmanowa-Petrusewicz I. A collaborative study on the natural history of childhood and juvenile onset proximal spinal muscular atrophy (type II and III SMA): 569 patients.J Neurol Sci. 1997; 146: 67-72
- Content validity and clinical meaningfulness of the HFMSE in spinal muscular atrophy.BMC Neurol. 2017; 17: 39
- Nusinersen in adults with 5q spinal muscular atrophy: a non-interventional, multicentre, observational cohort study.Lancet Neurol. 2020; 19: 317-325
- 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: evidence of sustained clinical improvement and lessened fatigue in older ambulatory patients with spinal muscular atrophy.Muscle Nerve. 2020; 61: 1-2
- Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy.N Engl J Med. 2018; 378: 625-635
- Nusinersen for older patients with spinal muscular atrophy: a real-world clinical setting experience.Muscle Nerve. 2020; 61: 222-226
- Drug treatment for spinal muscular atrophy types II and III.Cochrane Database Syst Rev. 2020; 1CD006282
- Safety and Treatment Effects of Nusinersen in Longstanding Adult 5q-SMA Type 3 - A Prospective Observational Study.J Neuromuscul Dis. 2019; 6: 453-465
- Prospective Cohort Study of Nusinersen Treatment in Adults with Spinal Muscular Atrophy.J Neuromuscul Dis. 2020;
- Different trajectories in upper limb and gross motor function in spinal muscular atrophy.Muscle Nerve. 2021;
- Spinal muscular atrophy functional composite score: a functional measure in spinal muscular atrophy.Muscle Nerve. 2015; 52: 942-947