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Volumn 61, Issue 10, 2006, Pages 593-598

Experience with the enzyme substitution therapy in mucopolysaccharidosis;Skúsenosti s enzýmovou substitučnou liečbou u mukopolysacharidózy

Author keywords

Enzyme substitution therapy; Fabry disease; Gaucher diseases; Lysosomal diseases; Mucopolysaccharidosis type I and type VI; Pompe disease

Indexed keywords

LARONIDASE;

EID: 33750204361     PISSN: 00692328     EISSN: None     Source Type: Journal    
DOI: None     Document Type: Article
Times cited : (1)

References (11)
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    • Anderson-Fabry disease: Clinical manifestations and impact of disease in a cohort of 60 obligate carrier females
    • MacDermot KD, Holmes A, Miners AH. Anderson-Fabry disease: clinical manifestations and impact of disease in a cohort of 60 obligate carrier females. J. Med. Genet. 2001;38:769-775.
    • (2001) J. Med. Genet. , vol.38 , pp. 769-775
    • MacDermot, K.D.1    Holmes, A.2    Miners, A.H.3
  • 2
    • 33750202744 scopus 로고    scopus 로고
    • The mucopolysaccharidoses
    • Zimran A. (ed). UK: Adis International Ltd.
    • Walkley SU. The mucopolysaccharidoses. In: Zimran A. (ed). Glycolipid Storage Disorders. UK: Adis International Ltd., 2004:101-111.
    • (2004) Glycolipid Storage Disorders , pp. 101-111
    • Walkley, S.U.1
  • 6
    • 0035153479 scopus 로고    scopus 로고
    • Lessons learned from the development of enzyme therapy for Gaucher disease
    • Barranger JA, O'Rourke E. Lessons learned from the development of enzyme therapy for Gaucher disease. J. Inherit. Metab. Dis. 2001;24(Suppl. 2):89-96.
    • (2001) J. Inherit. Metab. Dis. , vol.24 , Issue.SUPPL. 2 , pp. 89-96
    • Barranger, J.A.1    O'Rourke, E.2
  • 7
    • 0037177166 scopus 로고    scopus 로고
    • Prevalence of Anderson-Fabry disease in male patients with late onset hypertrophic cardiomyopathy
    • Sachdev B, Takenaka T, Teraguchi H. Prevalence of Anderson-Fabry disease in male patients with late onset hypertrophic cardiomyopathy. Circulation. 2002;105:1407-1411.
    • (2002) Circulation , vol.105 , pp. 1407-1411
    • Sachdev, B.1    Takenaka, T.2    Teraguchi, H.3
  • 8
    • 0035097499 scopus 로고    scopus 로고
    • A phase I/II clinical trial of enzyme replacement in Fabry disease: Pharmacokinetic, substrate clearance, and safety studies
    • Eng CM, Banikazemi M, Gordon RE. A phase I/II clinical trial of enzyme replacement in Fabry disease: pharmacokinetic, substrate clearance, and safety studies. Amer. J. Hum. Genet. 2001;68:711-722.
    • (2001) Amer. J. Hum. Genet. , vol.68 , pp. 711-722
    • Eng, C.M.1    Banikazemi, M.2    Gordon, R.E.3
  • 9
    • 27744493202 scopus 로고    scopus 로고
    • Direct comparison of measures of endurance, mobility, and joint function during enzym-replacement therapy of mucopolysaccharidosis VI (Maroteaux-Lamy syndrome): Results after 48 weeks in a phase 2 Open-Label Clinical Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase
    • Harmatz P, Ketteridge D, et al. Direct comparison of measures of endurance, mobility, and joint function during enzym-replacement therapy of mucopolysaccharidosis VI (Maroteaux-Lamy syndrome): Results after 48 weeks in a phase 2 Open-Label Clinical Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase. Pediatrics. 2005;115:681-749.
    • (2005) Pediatrics , vol.115 , pp. 681-749
    • Harmatz, P.1    Ketteridge, D.2
  • 11
    • 21144449402 scopus 로고    scopus 로고
    • Disease severity in children and adults with Pompe disease related to age and disease duratio
    • Hagemans MLC, Winkel LPF, et al. Disease severity in children and adults with Pompe disease related to age and disease duratio. Neurology. 2005;64:2139-2141.
    • (2005) Neurology , vol.64 , pp. 2139-2141
    • Hagemans, M.L.C.1    Winkel, L.P.F.2


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.