Enzyme replacement therapy and/or hematopoietic stem cell transplantation at diagnosis in patients with mucopolysaccharidosis type I: results of a European consensus procedure

by Sykora Karl-Walter, Rovelli Attilio, Parini Rossella, O'Meara Anne, Offringa Martin, Mengel Eugen, Mahlaoui Nizar, van der Lee Johanna H, Jones Simon A, Das Anibh M, Boelens Jaap J, de Ru Minke H, Valayannopoulos Vassili, Vellodi Ashok, Wynn Robert F, Wijburg Frits A
Published 2011-08-01