Transplantation in inborn errors of metabolism: Current considerations and future perspectives

British Journal of Haematology

Volumn 167, Issue 3, 2014, Pages 293-303

  Boelens, Jaap Jan ^a   Orchard, Paul J ^b   Wynn, Robert F ^c  

^a UNIVERSITY MEDICAL CENTER UTRECHT   (Netherlands)

^b UNIVERSITY OF MINNESOTA   (United States)

^c ROYAL MANCHESTER CHILDREN S HOSPITAL   (United Kingdom)

Author keywords

Inborn errors; Metabolism; Transplantation

Indexed keywords

ADJUVANT THERAPY; ADRENOLEUKODYSTROPHY; ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION; COMBINATION CHEMOTHERAPY; DISEASE COURSE; DISORDERS OF PEROXISOMAL FUNCTIONS; ENGRAFTMENT; ENZYME REPLACEMENT; GENE THERAPY; HUMAN; HURLER SYNDROME; INBORN ERROR OF METABOLISM; LYSOSOME STORAGE DISEASE; METACHROMATIC LEUKODYSTROPHY; MYELOABLATIVE CONDITIONING; NEWBORN SCREENING; PHENOTYPE; PRIORITY JOURNAL; REVIEW; SURVIVAL RATE; TIME TO TREATMENT; TREATMENT INDICATION; TREATMENT OUTCOME; TREATMENT RESPONSE; ALLOGRAFT; BONE MARROW TRANSPLANTATION; EARLY DIAGNOSIS; FORECASTING; GRAFT VS HOST DISEASE; METABOLISM, INBORN ERRORS; MULTIMODALITY CANCER THERAPY; NEWBORN; PATIENT SELECTION; QUALITY OF LIFE; STEM CELL TRANSPLANTATION; TIME;

IMMUNOSUPPRESSIVE AGENT;

ALLOGRAFTS; BONE MARROW TRANSPLANTATION; COMBINED MODALITY THERAPY; EARLY DIAGNOSIS; ENZYME REPLACEMENT THERAPY; FORECASTING; GENETIC THERAPY; GRAFT VS HOST DISEASE; HUMANS; IMMUNOSUPPRESSIVE AGENTS; INFANT, NEWBORN; METABOLISM, INBORN ERRORS; NEONATAL SCREENING; PATIENT SELECTION; QUALITY OF LIFE; STEM CELL TRANSPLANTATION; TIME FACTORS;

EID: 84912002256     PISSN: 00071048     EISSN: 13652141     Source Type: Journal    
DOI: 10.1111/bjh.13059     Document Type: Review

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