Parental experience of enzyme replacement therapy for hunter syndrome

Irish Medical Journal

Volumn 106, Issue 4, 2013, Pages

  Buraczewska, M ^a   O'Leary, Daire ^a   Walsh, Olivia ^a   Monavari, Ahmad ^a   Crushell, Ellen ^a  

^a CHILDREN S UNIVERSITY HOSPITAL   (Ireland)

Author keywords

[No Author keywords available]

Indexed keywords

GLYCOSAMINOGLYCAN; IDURONATE 2 SULFATASE;

ADOLESCENT; ARTICLE; ATTENTION DEFICIT DISORDER; CHILD; CLINICAL ARTICLE; DISEASE ASSOCIATION; EMPLOYMENT STATUS; ENZYME REPLACEMENT; ETHNIC GROUP; FACIES; FEMALE; HAIR; HEART VENTRICLE HYPERTROPHY; HEPATOSPLENOMEGALY; HOME CARE; HOSPITALIZATION; HUMAN; HUNTER SYNDROME; IRELAND; IRISH; MALE; PARENTAL ATTITUDE; PARENTAL BEHAVIOR; PRESCHOOL CHILD; QUALITY OF LIFE; QUESTIONNAIRE; RESPIRATORY TRACT INFECTION; RETROSPECTIVE STUDY; SCHOOL CHILD; SKIN; SLEEP DISORDERED BREATHING; URINARY EXCRETION; ATTITUDE; LETTER; PARENT; TIME;

ADOLESCENT; ATTITUDE; CHILD; CHILD, PRESCHOOL; ENZYME REPLACEMENT THERAPY; FEMALE; HOME CARE SERVICES; HUMANS; IDURONATE SULFATASE; IRELAND; MALE; MUCOPOLYSACCHARIDOSIS II; PARENTS; QUESTIONNAIRES; TIME FACTORS;

EID: 84876263788     PISSN: 03323102     EISSN: None     Source Type: Journal    
DOI: None     Document Type: Article

References (8)

1. Neufeld EF, Muenzer J. The mucopolysaccharidoses. In:Scriver CR, Beaudet AL, Sly WS, Valle D, editors. The metabolic and molecular bases of inherited disease. 8th ed. New York: McGraw. Hill, 2001, 3421.52.
2. Schwartz IV, Ribeiro MG, Mota JG, Toralles MB, Correia P, Horovitz D. A clinical study of 77 patients with mucopolysaccharidosis type II. Acta Paediatr 2007; 96;Suppl 455: 63.70.
3. Young ID, Harper PS, Archer IM, Newcombe RG. A clinical and genetic study of Hunter. s syndrome. 1. Heterogeneity. J Med Genet 1982; 19: 401.7.
4. J Edmond Wraith, Enzyme replacement therapy with idursulfase in patients with mucopolysaccharidosis type II. Acta Paediatr 2008; Volume 97, Suppl 457, pages 76.7
5. Burton BK, Guffon N, Roberts J, van der Ploeg AT, Jones SA; HOS investigators. Home treatment with intravenous enzyme replacement therapy with idursulfase for mucopolysaccharidosis type II-data from the Hunter Outcome Survey. Mol Genet Metab. 2010;101:123-9.
6. Milligan A, Hughes D, Goodwin S, Richfield L, Mehta A. Intravenous enzyme replacement therapy: better in home or hospital? Br J Nurs. 2006 Mar 23-Apr 12;15:330-3.
7. Schulze-Frenking G, Jones SA, Roberts J, Beck M, Wraith JE, Effects of enzyme replacement therapy on growth in patients with mucopolysaccharidosis type II. J Inherit Metab Dis 2011; 34:203-208
8. Burton BK, Wiesman C, Paras A, Kim K, Katz R. Home infusion therapy is safe and enhances compliance in patients with mucopolysaccharidoses. Mol Genet Metab. 2009 Jul;97:234-6. Epub 2009 Apr 21