Early treatment is associated with improved cognition in Hurler syndrome

Neurodevelopmental Outcomes of Umbilical Cord Blood Transplantation in Metachromatic Leukodystrophy

(2013) Holly R. Martin et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

Hurler disease (mucopolysaccharidosis type IH): clinical features and consanguinity in Tunisian population

(2012) Latifa Chkioua et al.   Diagnostic Pathology

Enzyme Replacement is Associated with Better Cognitive Outcomes after Transplant in Hurler Syndrome

(2012) Julie B. Eisengart et al.   JOURNAL OF PEDIATRICS

An exploratory study of brain function and structure in mucopolysaccharidosis type I: Long term observations following hematopoietic cell transplantation (HCT)

(2012) Elsa Shapiro et al.   MOLECULAR GENETICS AND METABOLISM

Mucopolysaccharidosis I mutations in Chinese patients: identification of 27 novel mutations and 6 cases involving prenatal diagnosis

(2011) X Wang et al.   CLINICAL GENETICS

IDUA mutational profiling of a cohort of 102 European patients with mucopolysaccharidosis type I: identification and characterization of 35 novel α-L-iduronidase (IDUA) alleles

(2011) Francesca Bertola et al.   HUMAN MUTATION

Variable disease progression after successful stem cell transplantation: Prospective follow-up investigations in eight patients with Hurler syndrome

(2011) Lorenz Grigull et al.   PEDIATRIC TRANSPLANTATION

Mucopolysaccharidosis type I in 21 Czech and Slovak patients: Mutation analysis suggests a functional importance of C-terminus of the IDUA protein

(2009) Alzbeta Vazna et al.   AMERICAN JOURNAL OF MEDICAL GENETICS PART A

Intravenous and Intrathecal Enzyme Replacement Before and After Hematopoietic Cell Transplantation for Hurler Syndrome

(2009) J. Tolar et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

Pre-transplant risk factors affecting outcome in Hurler syndrome

(2009) P J Orchard et al.   BONE MARROW TRANSPLANTATION

Outcome in six children with mucopolysaccharidosis type IH, hurler syndrome, after haematopoietic stem cell transplantation (HSCT)

(2008) Gunilla Malm et al.   ACTA PAEDIATRICA

The Clinical Outcome of Hurler Syndrome after Stem Cell Transplantation

(2008) Mieke Aldenhoven et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

Unrelated donor umbilical cord blood transplantation for inherited metabolic disorders in 159 pediatric patients from a single center: influence of cellular composition of the graft on transplantation outcomes

(2008) V. K. Prasad et al.   BLOOD

Cognitive and Academic Consequences of Stem-Cell Transplantation in Children

(2008) Sean Phipps et al.   JOURNAL OF CLINICAL ONCOLOGY