SMA is a disease that will keep on progressing without proper intervention.1 SMA impacts the lives of patients, gradually making them more and more reliant on others to do small things for them – bringing their lives to a standstill.
The characters shown are real patients and the required consent to use their stories has been obtained from the patients and families. Photographs are for illustrative purposes only.
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LIVES COME TO A STANDSTILL WITH SMA
SMA is a debilitating neuromuscular disease characterised by motor neuron degeneration and loss of muscle strength.2 As the disease progresses, patients face the gradual erosion of pulmonary and motor functions to the point where even simple tasks become impossible.1,3-5
Given that prospect, any treatment that can stabilise disease progression will be welcomed as a therapeutic breakthrough.5
The natural history of SMA disease
79 patients declined by an average of -1.71 points over 36 months on the HFMSE scale.1
Adapted from Kaufmann et. al.1
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For people living with SMA, care outcomes today are better than they have ever been. Find out more about disease stabilisation.
The characters shown are real patients and the required consent to use their stories has been obtained from the patients and families. Photographs are for illustrative purposes only.
References
1. Kaufmann P, McDermott MP, Darras BT, et al. Prospective cohort study of spinal muscular atrophy types 2 and 3. Neurology 2012;79(18):1889-1897.
2. Lunn MR, Wang CH. Spinal muscular atrophy. Lancet 2008;371:2120-2133.
3. Wadman RI, Wijngaarde CA, Stam M, et al. Muscle strength and motor function throughout life in a cross-sectional cohort of 180 patients with SMA types 1c-4. Eur J Neurol 2018;25(3):512-518.
4. Paradis AD, Wassel CL, Dreyfus J, et al. Symptoms and Complications Among Later Childhood, Adolescent, and Adult Spinal Muscular Atrophy Patients: A Natural History Study Within US Hospitals. Poster P206 presented at MDA 2019; Orlando, Florida, USA.
5. Rouault F, Christie-Brown V, Broekgaarden R, et al. Disease impact on general well-being and therapeutic expectations of European Type II and Type III spinal muscular atrophy patients. Neuromuscul Disord 2017;27(5):428-438.
