SMA CLINICAL DATA, OUTCOME MEASURES AND REGISTRIES

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Spinal muscular atrophy (SMA) is a rare genetic, life-threatening neuromuscular disease. Data on the cost burden of SMA type 1 (SMA1) for U.S. health plans are limited as most studies do not differentiate between SMA types or have consisted of a post-hoc subset based on age at diagnosis. This retrospective analysis estimated the economic burden of SMA1 using QuintilesIMS’s PharMetrics Plus Health Plan Claims Database. Infants with ICD-9 codes for SMA ≤1 year old were classified as SMA1 (N=119) and matched (1:1) with a random sample of infants by age, gender, index year, and Charlson Comorbidity Index (CCI).

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