Age at symptom onset, diagnosis, and enzyme replacement therapy (ERT) initiation among family members with mucopolysaccharidosis II (Hunter Syndrome): Data from the Hunter Outcome Survey (HOS)

作者:Ficicioglu Can; Mendelsohn Nancy J; Giugliani Roberto; Lampe Christina; Fang Juanzhi
来源:9th Annual World Symposium of the Lysosomal-Disease-Network (LDN), 2013-02-12 to 2013-02-15.
DOI:10.1016/j.ymgme.2012.11.084