Patient and caregiver preferences for treatment attributes in type 2 and non-ambulatory type 3 spinal muscular atrophy: a pan-european stated preference survey

S. H. Lo, C. Lawrence, Y. Marti-Gil, A. Cafe,A. Lloyd

VALUE IN HEALTH（2022）

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摘要

Spinal muscular atrophy (SMA) is a rare, genetic neuromuscular disease. The study objective was to evaluate patient and caregiver preferences for treatment attributes in Type 2 and non-ambulatory Type 3 SMA across five European countries using a stated preference survey.

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关键词

spinal muscular atrophy,treatment attributes,caregiver preferences,type,non-ambulatory,pan-european

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