J Inherit Metab Dis. 1992;15(6):911-8.

Bone marrow transplantation for Sanfilippo disease type B.

Vellodi A, Young E, New M, Pot-Mees C, Hugh-Jones K.

Source

Bone Marrow Transplant Unit, Westminster Children's Hospital, London, UK.

Abstract

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 alpha-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 alpha-glucosaminidase activity and urinary glycosaminoglycan excretion are reported.

PMID:: 1293388; [PubMed - indexed for MEDLINE]

Publication Types, MeSH Terms, Substances

Publication Types

Case Reports

MeSH Terms

Substances

LinkOut - more resources

Supplemental Content

Save items

Related citations in PubMed

Long-term outcome of Hurler syndrome following bone marrow transplantation. [Am J Med Genet. 1993]
Long-term outcome of Hurler syndrome following bone marrow transplantation.
Whitley CB, Belani KG, Chang PN, Summers CG, Blazar BR, Tsai MY, Latchaw RE, Ramsay NK, Kersey JH. Am J Med Genet. 1993 Apr 15; 46(2):209-18.
Review Sanfilippo disease type B. A case report and review of the literature on recent advances in bone marrow transplantation. [Turk J Pediatr. 1995]
Review Sanfilippo disease type B. A case report and review of the literature on recent advances in bone marrow transplantation.
Güngör N, Tunçbilek E. Turk J Pediatr. 1995 Apr-Jun; 37(2):157-63.
The laboratory diagnosis of Sanfilippo disease. [Clin Chim Acta. 1977]
The laboratory diagnosis of Sanfilippo disease.
Whiteman P, Young E. Clin Chim Acta. 1977 Apr 1; 76(1):139-47.
Sanfilippo B disease in two related sibships. Biochemical studies in patients, parents and sibs. [Clin Genet. 1976]
Sanfilippo B disease in two related sibships. Biochemical studies in patients, parents and sibs.
Liem KO, Giesberts MA, van de Kamp JJ, van Pelt JF, Hooghwinkel GJ. Clin Genet. 1976 Nov; 10(5):273-8.
Review Bone marrow transplantation in Hunter syndrome (mucopolysaccharidosis type II): two-year follow-up of the first Italian patient and review of the literature. [Pediatr Med Chir. 1995]
Review Bone marrow transplantation in Hunter syndrome (mucopolysaccharidosis type II): two-year follow-up of the first Italian patient and review of the literature.
Coppa GV, Gabrielli O, Zampini L, Pierani P, Giorgi PL, Jezequel AM, Orlandi F, Miniero R, Busca A, De Luca T. Pediatr Med Chir. 1995 May-Jun; 17(3):227-35.

See reviews... See all...

Cited by 2 PubMed Central articles

Growth patterns and the use of growth hormone in the mucopolysaccharidoses. [J Pediatr Rehabil Med. 2010]
Growth patterns and the use of growth hormone in the mucopolysaccharidoses.
Polgreen LE, Miller BS. J Pediatr Rehabil Med. 2010; 3(1):25-38.
Correction of mucopolysaccharidosis type IIIb fibroblasts by lentiviral vector-mediated gene transfer. [Biochem J. 2002]
Correction of mucopolysaccharidosis type IIIb fibroblasts by lentiviral vector-mediated gene transfer.
Villani GR, Follenzi A, Vanacore B, Di Domenico C, Naldini L, Di Natale P. Biochem J. 2002 Jun 15; 364(Pt 3):747-53.

Related information

Related Citations
Calculated set of PubMed citations closely related to the selected article(s) retrieved using a word weight algorithm. Related articles are displayed in ranked order from most to least relevant, with the “linked from” citation displayed first.
Gene (OMIM)
Gene records associated with Online Mendelian Inheritance in Man (OMIM) records that cite the current articles in their reference lists.
Nucleotide (Weighted)
Nucleotide records associated with the current articles through the Gene database. These are the related sequences on the Gene record that are added manually by NCBI.
OMIM (calculated)
Online Mendelian Inheritance in Man (OMIM) records that include the current articles as references in the light bulb links within or in the citations at the end of the OMIM record. The references available through the light bulb link are collected using the PubMed related articles algorithm to identify records with similar terminology to the OMIM record.
OMIM (cited)
Online Mendelian Inheritance in Man (OMIM) records that include the current articles as reference cited at the end of the OMIM record.
Protein (Weighted)
Protein records associated with the current articles through related Gene database records. These are the related sequences on the Gene record that are added manually by NCBI.
Substance (MeSH Keyword)
PubChem chemical substance (submitted) records that are classified under the same Medical Subject Headings (MeSH) controlled vocabulary as the current articles.
Cited in PMC
Full-text articles in the PubMed Central Database that cite the current articles.
Cited in Books
NCBI Bookshelf books that cite the current articles.

Recent activity

Clear Turn Off Turn On

Bone marrow transplantation for Sanfilippo disease type B.
Bone marrow transplantation for Sanfilippo disease type B.
J Inherit Metab Dis. 1992 ;15(6):911-8.

PubMed
Both the Fas ligand and inducible nitric oxide synthase are needed for control o...
Both the Fas ligand and inducible nitric oxide synthase are needed for control of parasite replication within lesions in mice infected with Leishmania major whereas the contribution of tumor necrosis factor is minimal.
Infect Immun. 2003 Sep ;71(9):5287-95.

PubMed
Hereditary xanthinuria and Ehlers-Danlos syndrome.
Hereditary xanthinuria and Ehlers-Danlos syndrome.
J Inherit Metab Dis. 1992 ;15(6):881-2.

PubMed
Skin facilitates Candida albicans mating.
Skin facilitates Candida albicans mating.
Infect Immun. 2003 Sep ;71(9):4970-6.

PubMed
Glycogen debranching enzyme deficiency: long-term study of serum enzyme activiti...
Glycogen debranching enzyme deficiency: long-term study of serum enzyme activities and clinical features.
J Inherit Metab Dis. 1992 ;15(6):869-81.

PubMed

Your browsing activity is empty.

Activity recording is turned off.

Turn recording back on

PubMed

Result Filters

Display Settings:

Send to:

Bone marrow transplantation for Sanfilippo disease type B.

Source

Abstract

Publication Types, MeSH Terms, Substances

Publication Types

MeSH Terms

Substances

LinkOut - more resources

Full Text Sources

Other Literature Sources

Medical

Supplemental Content

Save items

Related citations in PubMed

Cited by 2 PubMed Central articles

Related information

Recent activity

Simple NCBI Directory

Getting Started

Resources

Popular

Featured

NCBI Information