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Disease progression in SMA

The natural course of spinal muscular atrophy is characterised by progressive muscle atrophy and loss of motor function. Stabilisation of function is important to many people living with SMA1,2

Disease progression in SMA may be divided into 3 conceptual phases: preclinical, subacute, and chronic2

ACQUISITION OF GROSS MOTOR MILESTONES IN TYPICALLY DEVELOPING CHILDREN COMPARED WITH INDIVIDUALS WITH SMA2
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Adapted from Swoboda et al. 2007.2
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Loss of function in later-onset SMA may have a major impact on quality of life

In a European survey of 822 individuals (8-73 years of age) with later-onset (Type II and Type III) SMA, a subgroup of patients identified their current functional abilities that if lost would affect their quality of life (QoL):3

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97% of the respondents with later-onset (Types II and III) SMA reported that stabilisation of the level of function would represent progress.3

Stabilisation of functional abilities may be important to individuals with later-onset SMA

In the same survey, individuals with later-onset SMA identified those functional abilities that they have retained and would most want to stabilise.† These included:3

  • Feeding him/herself
  • Washing independently
  • Using restroom independently
  • Performing transfers independently
  • Using a keyboard
  • Turning in bed
  • Writing with a pen
  • Brushing his/her teeth
  • Dressing by him/herself
  • Brushing his/her hair
†Participants were asked to choose the 3 functions, ranked 1 through 3, in decreasing order of priority they would most like to stabilise.
References
  1. Darras BT et al. Neuromuscular Disorders of Infancy, Childhood, and Adolescence: A Clinician’s Approach. 2nd ed. London, UK: Elsevier; 2015.
  2. Swoboda KJ et al. J Child Neurol 2007; 22: 957–66.
  3. Rouault F et al. Neuromuscul Disord 2017; 27: 428–38.