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Mucopolysaccharidosis type II (Hunter syndrome): a clinical review and recommendations for treatment in the era of enzyme replacement therapy
10 auth.
J. E. Wraith,
M. Scarpa,
M. Beck,
O. Bodamer,
L. de Meirleir,
N. Guffon,
...
A. Meldgaard Lund,
G. Malm,
A. T. van der Ploeg,
J. Zeman
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8 |
2007 |
8
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Sustained, long-term renal stabilization after 54 months of agalsidase beta therapy in patients with Fabry disease.
12 auth.
D. Germain,
S. Waldek,
M. Banikazemi,
D. Bushinsky,
J. Charrow,
R. Desnick,
...
P. Lee,
T. Loew,
A. Vedder,
R. Abichandani,
W. Wilcox,
N. Guffon
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8 |
2007 |
8
๐ข
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Long-term safety and efficacy of enzyme replacement therapy for Fabry disease.
8 auth.
William R. Wilcox,
M. Banikazemi,
N. Guffon,
Steve Waldek,
Philip Lee,
G. Linthorst,
...
Robert J. Desnick,
Dominique P. Germain
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8 |
2004 |
8
๐
|
|
๐
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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.
17 auth.
P. Harmatz,
R. Giugliani,
I. Schwartz,
N. Guffon,
E. Teles,
M. C. S. Miranda,
J. E. Wraith,
M. Beck,
Laila Arash,
M. Scarpa,
...
ZiโFan Yu,
J. Wittes,
K. Berger,
M. Newman,
A. Lowe,
E. Kakkis,
S. Swiedler
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8 |
2006 |
8
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Ten-year outcome of enzyme replacement therapy with agalsidase beta in patients with Fabry disease
15 auth.
D. Germain,
J. Charrow,
R. Desnick,
Genzyme Judy Kempf,
Sanofi company,
R. Lachmann,
Genzyme Roberta Lemay,
G. Linthorst,
S. Packman,
C. Scott,
...
N. Guffon,
S. Waldek,
D. Warnock,
N. Weinreb,
W. Wilcox
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8 |
2015 |
8
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๐ข
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Enzyme Replacement Therapy in Patients Who Have Mucopolysaccharidosis I and Are Younger Than 5 Years: Results of a Multinational Study of Recombinant Human ฮฑ-l-Iduronidase (Laronidase)
8 auth.
J. E. Wraith,
M. Beck,
R. Lane,
A. T. van der Ploeg,
E. Shapiro,
Yong Xue,
...
E. Kakkis,
N. Guffon
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7 |
2007 |
7
๐ข
|
|
๐
|
Safety and efficacy of recombinant human alpha-galactosidase A replacement therapy in Fabry's disease.
9 auth.
C. Eng,
N. Guffon,
W. Wilcox,
Dominique P. Germain,
P. Lee,
S. Waldek,
...
L. Caplan,
G. Linthorst,
R. Desnick
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7 |
2001 |
7
๐
|
|
๐
|
Outcome of 27 patients with Hurler's syndrome transplanted from either related or unrelated haematopoietic stem cell sources
18 auth.
G. Souillet,
N. Guffon,
I. Maire,
M. Pujol,
P. Taylor,
F. Sevin,
N. Bleyzac,
C. Mulier,
A. Durin,
K. Kรฉbaili,
...
C. Galambrun,
Y. Bertrand,
R. Froissart,
C. Dorche,
L. Gebuhrer,
C. Garin,
J. Bรฉrard,
P. Guibaud
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7 |
2003 |
7
๐
|
|
๐
|
Direct Comparison of Measures of Endurance, Mobility, and Joint Function During Enzyme-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
9 auth.
P. Harmatz,
D. Ketteridge,
R. Giugliani,
N. Guffon,
E. Teles,
M. C. S. Miranda,
...
ZiโFan Yu,
S. Swiedler,
J. Hopwood
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7 |
2005 |
7
๐
|
|
๐
|
Long-term follow-up of endurance and safety outcomes during enzyme replacement therapy for mucopolysaccharidosis VI: Final results of three clinical studies of recombinant human N-acetylgalactosamine 4-sulfatase.
19 auth.
P. Harmatz,
R. Giugliani,
Ida V. D. Schwartz,
N. Guffon,
E. Teles,
M. C. S. Miranda,
J. E. Wraith,
M. Beck,
Laila Arash,
M. Scarpa,
...
D. Ketteridge,
J. Hopwood,
B. Plecko,
R. Steiner,
C. Whitley,
P. Kaplan,
ZiโFan Yu,
S. Swiedler,
C. Decker
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7 |
2008 |
7
๐
|
|
๐
|
Mucopolysaccharidosis type II: European recommendations for the diagnosis and multidisciplinary management of a rare disease
26 auth.
M. Scarpa,
Z. Almรกssy,
M. Beck,
O. Bodamer,
I. Bruce,
L. de Meirleir,
N. Guffon,
E. Guillรฉn-Navarro,
P. Hensman,
Simon A. Jones,
W. Kamin,
C. Kampmann,
C. Lampe,
C. Lavery,
E. Leรฃo Teles,
...
B. Link,
A. Lund,
G. Malm,
S. Pitz,
M. Rothera,
C. Stewart,
A. Tylki-Szymaลska,
A. T. van der Ploeg,
R. Walker,
J. Zeman,
J. E. Wraith
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7 |
2011 |
7
๐
|
