Newborn screening for lysosomal storage disorders
- 31 August 2006
- journal article
- Published by Elsevier in Molecular Genetics and Metabolism
- Vol. 88 (4) , 307-314
- https://doi.org/10.1016/j.ymgme.2006.02.013
Abstract
No abstract availableKeywords
This publication has 36 references indexed in Scilit:
- Transplantation of Umbilical-Cord Blood in Babies with Infantile Krabbe's DiseaseNew England Journal of Medicine, 2005
- Cord-Blood Transplants from Unrelated Donors in Patients with Hurler's SyndromeNew England Journal of Medicine, 2004
- Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human α-L-iduronidase (laronidase)The Journal of Pediatrics, 2004
- Agalsidase alfa: specific treatment for Fabry diseaseHospital Medicine, 2002
- Agalsidase alfa – a preparation for enzyme replacement therapy in Anderson–Fabry diseaseExpert Opinion on Investigational Drugs, 2002
- Wolman disease successfully treated by bone marrow transplantationBone Marrow Transplantation, 2000
- Prevalence of Lysosomal Storage DisordersJAMA, 1999
- Allogeneic bone marrow transplantation for lysosomal storage diseasesThe Lancet, 1995
- Long‐term clinical progress in bone marrow transplanted mucopolysaccharidosis type I patients with a defined genotypeJournal of Inherited Metabolic Disease, 1993
- Replacement Therapy for Inherited Enzyme Deficiency — Macrophage-Targeted Glucocerebrosidase for Gaucher's DiseaseNew England Journal of Medicine, 1991