Figure 1

Results of literature search. Flow diagram demonstrates the review and selection process for published articles and gray-literature documents identified by the original SER (through 31 December 2015). aThe ERT box includes 25 studies, 2 SERs, 7 studies on MPSII prevalence and phenotypes, 4 on very early ERT, and 9 on other topics. The 11 gray-literature documents include the Shire package insert, 2 US Food and Drug Administration reports, and 8 gray documents included in Supplementary Table S10. bGenotype–phenotype association data will be reported in a separate paper in preparation. ERT, enzyme replacement therapy; HSCT, hematopoietic stem cell transplantation; SER, systematic evidence review.