Biochemical monitoring after haemopoietic stem cell transplant for Hurler syndrome (MPSIH): implications for functional outcome after transplant in metabolic disease

Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human α-L-iduronidase (laronidase)

J. E. Wraith, L. Clarke, Michael Beck et al. · The Journal of Pediatrics · 2004 · 546 citations

Life Sciences, Biochemistry, Enzymology +7

Cord-Blood Transplants from Unrelated Donors in Patients with Hurler's Syndrome

Susan Staba, Maria L. Escolar, Michele D. Poe et al. · New England Journal of Medicine · 2004 · 481 citations · Full text

Medicine, Hematology, Pediatrics +17

Rapid typing of tandemly repeated hypervariable loci by the polymerase chain reaction: application to the apolipoprotein B 3' hypervariable region.

Eric Boerwinkle, WanLong Xiong, Eric Fourest et al. · Proceedings of the National Academy of Sciences · 1989 · 473 citations

Life Sciences, Genetics, Molecular Genetics +16

Hurler and Hunter Syndromes: Mutual Correction of the Defect in Cultured Fibroblasts

Joseph C. Fratantoni, Clara W. Hall, Elizabeth F. Neufeld · Science · 1968 · 442 citations

Life Sciences, Genetics, Molecular Genetics +8

Hurler Syndrome: II. Outcome of HLA-Genotypically Identical Sibling and HLA-Haploidentical Related Donor Bone Marrow Transplantation in Fifty-Four Children

Charles Peters, Elsa Shapiro, James R. Anderson et al. · Blood · 1998 · 340 citations · Full text

Medicine, Hematology, Immunology +16