Subject Areas on Research
- Cord blood is the optimal graft source for the treatment of pediatric patients with lysosomal storage diseases: clinical outcomes and future directions.
- Immune response to enzyme replacement therapies in lysosomal storage diseases and the role of immune tolerance induction.
- Immunomodulatory gene therapy in lysosomal storage disorders.
- Infantile sialic acid storage disease: a rare cause of cytoplasmic vacuolation in pediatric patients.
- Multiplex newborn screening for Pompe, Fabry, Hunter, Gaucher, and Hurler diseases using a digital microfluidic platform.
- Newborn Screening for Lysosomal Storage Disorders: Quo Vadis?
- Newborn screening for lysosomal storage disorders.
- Posttransplant autoimmune hemolytic anemia and other autoimmune cytopenias are increased in very young infants undergoing unrelated donor umbilical cord blood transplantation.
- Rapid and effective screening for lysosomal storage disease: how close are we?
- Results of the cord blood transplantation study (COBLT): outcomes of unrelated donor umbilical cord blood transplantation in pediatric patients with lysosomal and peroxisomal storage diseases.
- The use of dried blood spot samples in the diagnosis of lysosomal storage disorders--current status and perspectives.
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