SCOPUS 정보 검색 플랫폼

New England Journal of Medicine

Volumn 345, Issue 1, 2001, Pages 55-57

New therapies for Fabry's disease

(1) Gahl, W A a

a NATIONAL INSTITUTES OF HEALTH (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ALPHA GALACTOSIDASE; GLOBOTRIAOSYLCERAMIDE;

EDITORIAL; ENZYME ACTIVE SITE; ENZYME BINDING; ENZYME REPLACEMENT; FABRY DISEASE; HUMAN; KIDNEY DISEASE; LYSOSOME STORAGE DISEASE; METABOLIC DISORDER; PRIORITY JOURNAL;

ALPHA-GALACTOSIDASE; FABRY DISEASE; GALACTOSE; HUMANS; TRIHEXOSYLCERAMIDES;

EID: 0035811740 PISSN: 00284793 EISSN: None Source Type: Journal
DOI: 10.1056/NEJM200107053450109 Document Type: Editorial

Times cited : (13)

References (12)

1
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- Desnick, R.J.¹ Ioannou, Y.A.² Eng, C.M.³

2
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- Safety and efficacy of recombinant human α-galactosidase A replacement therapy in Fabry's disease
- (2001) N Engl J Med , vol.345 , pp. 9-16
- Eng, C.M.¹ Guffon, N.² Wilcox, W.R.³

3
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- Improvement in cardiac function in the cardiac variant of Fabry's disease with galactose-infusion therapy
- (2001) N Engl J Med , vol.345 , pp. 25-32
- Frustaci, A.¹ Chimenti, C.² Ricci, R.³

4
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- Enzymatic defect in Fabry's disease: Ceramidetrihexosidase deficiency
- (1967) N Engl J Med , vol.276 , pp. 1163-1167
- Brady, R.O.¹ Gal, A.E.² Bradley, R.M.³ Martensson, E.⁴ Warshaw, A.L.⁵ Laster, L.⁶

5
- 0014932679
- Enzyme replacement in Fabry's disease, an inborn error of metabolism
- (1970) Science , vol.169 , pp. 987-989
- Mapes, C.A.¹ Anderson, K.L.² Sweeley, C.C.³ Desnick, R.J.⁴ Krivit, W.⁵

6
- 0035163539
- Fabry disease: Preclinical studies demonstrate the effectiveness of α-galactosidase A replacement in enzyme-deficient mice
- (2001) Am J Hum Genet , vol.68 , pp. 14-25
- Ioannou, Y.A.¹ Zeidner, K.M.² Gordon, R.E.³ Desnick, R.J.⁴

7
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- Infusion of alpha-galactosidase A reduces dssue globotriaosylceramide storage in patients with Fabry disease
- (2000) Proc Natl Acad Sci U S A , vol.97 , pp. 365-370
- Schifftmann, R.¹ Murray, G.J.² Treco, D.³

8
- 0035097499
- A phase 1/2 clinical trial of enzyme replacement in Fabry disease: Pharmacokinetic, substrate clearance, and safety studies
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- Eng, C.M.¹ Banikazemi, M.² Gordon, R.E.³

9
- 0000216808
- Gaucher disease
- Scriver CK, Beaudet AL, Sly WS, Valle D, eds. New York: McGraw-Hill
- (2001) The metabolic & molecular bases of inherited disease. 8th ed. , vol.3 , pp. 3635-3668
- Beutler, E.¹ Grabowski, G.A.²

10
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- Characterization of a mutant α-galactosidase gene product for the late-onset cardiac form of Fabry disease
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11
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12
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