caa a22 4500 469044195 CHVBK 20180323132819.0 cr unu---uuuuu 170328e19921101xx s 000 0 eng 10.1007/BF01800232 doi (NATIONALLICENCE)springer-10.1007/BF01800232 Bone marrow transplantation for Sanfilippo disease type B [Elektronische Daten] [A. Vellodi, E. Young, M. New, C. Pot-Mees, K. Hugh-Jones] Summary: Allogeneic bone marrow transplantation was performed on twins with Sanfilippo B disease. They were the first two patients with this disorder to undergo the procedure. There was definite evidence of engraftment as shown by conversion to donor blood group antigen and tissue type, and increased leukocyte α-glucosaminidase activity. Nine years post transplant, neither twin is as handicapped as her untreated brothers were at the same age, although in one twin hyperactivity and behavioural problems, characteristic of the disorder, are present. Details of the twins' intellectual development and growth, their α-glucosaminidase activity and urinary glycosaminoglycan excretion are reported. SSIEM and Kluwer Academic Publishers, 1992 Vellodi A. Bone Marrow Transplant Unit, Westminster Children's Hospital, Vincent Square, SW1P 2NS, London aut Young E. Division of Biochemistry and Metabolism, Institute of Child Health and Hospitals for Sick Children, 30 Guilford Street, WC1N 1EH, London, UK aut New M. Bone Marrow Transplant Unit, Westminster Children's Hospital, Vincent Square, SW1P 2NS, London aut Pot-Mees C. Bone Marrow Transplant Unit, Westminster Children's Hospital, Vincent Square, SW1P 2NS, London aut Hugh-Jones K. Bone Marrow Transplant Unit, Westminster Children's Hospital, Vincent Square, SW1P 2NS, London aut Journal of Inherited Metabolic Disease Kluwer Academic Publishers 15/6(1992-11-01), 911-918 0141-8955 15:6<911 1992 15 10545 https://doi.org/10.1007/BF01800232 text/html Onlinezugriff via DOI 1 research-article jats NATIONALLICENCE 856 40 https://doi.org/10.1007/BF01800232 text/html Onlinezugriff via DOI NATIONALLICENCE 700 1- Vellodi A. Bone Marrow Transplant Unit, Westminster Children's Hospital, Vincent Square, SW1P 2NS, London aut NATIONALLICENCE 700 1- Young E. Division of Biochemistry and Metabolism, Institute of Child Health and Hospitals for Sick Children, 30 Guilford Street, WC1N 1EH, London, UK aut NATIONALLICENCE 700 1- New M. Bone Marrow Transplant Unit, Westminster Children's Hospital, Vincent Square, SW1P 2NS, London aut NATIONALLICENCE 700 1- Pot-Mees C. Bone Marrow Transplant Unit, Westminster Children's Hospital, Vincent Square, SW1P 2NS, London aut NATIONALLICENCE 700 1- Hugh-Jones K. Bone Marrow Transplant Unit, Westminster Children's Hospital, Vincent Square, SW1P 2NS, London aut NATIONALLICENCE 773 0- Journal of Inherited Metabolic Disease Kluwer Academic Publishers 15/6(1992-11-01), 911-918 0141-8955 15:6<911 1992 15 10545 Metadata rights reserved Springer special CC-BY-NC licence nationallicence BK010053 XK010053 XK010000 NATIONALLICENCE NATIONALLICENCE NL-springer