Home treatment with elaprase® and Naglazyme® is safe in patients with mucopolysaccharidoses types II and VI, respectively

Journal of Inherited Metabolic Disease

Volumn 31, Issue 6, 2008, Pages 733-737

  Bagewadi, S ^a   Roberts, J ^a   Mercer, J ^a   Jones, S ^a   Stephenson, J ^b   Wraith, J E ^a  

^a ROYAL MANCHESTER CHILDREN S HOSPITAL   (United Kingdom)

^b NONE

Author keywords

[No Author keywords available]

Indexed keywords

GALSULFASE; IDURONATE 2 SULFATASE;

ADOLESCENT; ARTICLE; CHILD; CLINICAL ARTICLE; CLINICAL PROTOCOL; DRUG SAFETY; ENZYME REPLACEMENT; HOME CARE; HUMAN; HUNTER SYNDROME; MORQUIO SYNDROME; PATIENT SELECTION; TREATMENT PLANNING; VASCULAR ACCESS DEVICE;

ADOLESCENT; ANAPHYLAXIS; CHILD; CHILD, PRESCHOOL; HOME CARE SERVICES; HOME INFUSION THERAPY; HOME NURSING; HUMANS; IDURONATE SULFATASE; MUCOPOLYSACCHARIDOSIS II; MUCOPOLYSACCHARIDOSIS VI; N-ACETYLGALACTOSAMINE-4-SULFATASE; RECOMBINANT PROTEINS; TIME FACTORS; TREATMENT OUTCOME;

EID: 57649183911     PISSN: 01418955     EISSN: 15732665     Source Type: Journal    
DOI: 10.1007/s10545-008-0980-0     Document Type: Article

References (5)

1. Cox-Brinkman J, Timmermans RG, Wijburg FA, et al (2007) Home treatment with enzyme replacement therapy for mucopolysaccharidosis type I is feasible and safe. J Inherit Metab Dis. 30: 984. doi: 10.1007/ s10545-007-0686-8.
2. Harmatz P, Giugliani R, Schwartz I, et al (2006) Enzyme replacement therapy for mucopolysaccharidosis VI: A Phase 3, randomized, double-blind, placebo-controlled, multinational study of recombinant human N-acetylgalactosamine 4-sulfatase (recombinant human arylsulfatase B or RHASB), and follow on, open label extension study. J Pediatr 148: 533-539. doi: 10.1016/j.jpeds.2005.12.014.
3. Hughes DA, Milligan A, Mehta A (2007) Home therapy for lysosomal storage disorders. Br J Nurs 16: 1384, 1386-1389.
4. Milligan A, Hughes A, Goodwin S, Richfield L, Mehta A (2006) Intravenous enzyme replacement therapy: Better in home or hospital? Br J Nurs 15: 330-333.
5. Muenzer J, Wraith JE, Beck M, et al (2006) A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome). Genet Med 8: 465-473. doi: 10.1097/ 01.gim.0000232477.37660.fb.