Indirect estimation of the prevalence of spinal muscular atrophy Type I, II, and III in the United States

Abstract Background 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 unders...

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Principais autores:	Cathy Lally, Cynthia Jones, Wildon Farwell, Sandra P. Reyna, Suzanne F. Cook, W. Dana Flanders
Format:	Artigo
Jezik:	Inglês
Izdano:	BMC 2017-11-01
Serija:	Orphanet Journal of Rare Diseases
Teme:	Prevalence Spinal muscular atrophy Survival
Online dostop:	http://link.springer.com/article/10.1186/s13023-017-0724-z
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