Key result
Spinal muscular atrophy imposes a substantial economic burden, with an average total annual cost of $143,705 USD PPP per household, and significantly reduces quality of life for patients and caregivers.
Why the study?
The economic and health-related quality of life burden incurred by households with a child affected by spinal muscular atrophy needed to be quantified.
Observational (n=40)
Spinal muscular atrophy imposes a substantial economic and quality of life burden on households, which is essential for evaluating the cost-effectiveness of emerging therapies.
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SMA households face heavy costs and HRQoL losses; leaves open cost-effectiveness assessments of disease-modifying therapies.
Chambers et al. (2020) conducted an observational in Spinal muscular atrophy (n=40). Spinal muscular atrophy was evaluated on Economic and health-related quality of life (HRQoL) burden. Spinal muscular atrophy imposes a substantial economic burden, with an average total annual cost of $143,705 USD PPP per household, and significantly reduces quality of life for patients and caregivers.
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