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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patient
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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.