Indirect estimation of the prevalence of spinal muscular atrophy Type I, II, and III in the United States
Abstract
Spinal muscular atrophy (SMA) is a progressive, devastating disease and a leading inherited cause of infant mortality. The limited population-based literature is confined to small regional studies. Estimates of prevalence are needed to characterize the burden of SMA and to understand trends in prevalence by disease type as new treatments become available. The reported estimates of SMA genotype prevalence at birth consistently range from 8.5–10.3...
Paper Details
Title
Indirect estimation of the prevalence of spinal muscular atrophy Type I, II, and III in the United States
Published Date
Nov 28, 2017
Volume
12
Issue
1
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