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

Capturing phenotypic heterogeneity in MPS I: results of an international consensus procedure

by de Ru Minke H, Teunissen Quirine GA, van der Lee Johanna H, Beck Michael, Bodamer Olaf A, Clarke Lorne A, Hollak Carla E, Lin Shuan-Pei, Rojas Maria-Verónica, Pastores Gregory M, Raiman Julian A, Scarpa Maurizio, Treacy Eileen P, Tylki-Szymanska Anna, Wraith J Edmond, Zeman Jiri, Wijburg Frits A
Published 2012-04-01