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The Journal of Pediatrics
|
July 20, 2005
Pitfalls in newborn screening
Eric Crombez, Richard Koch, Stephen Cederbaum
American Journal of Hematology
|
April 24, 2015
Seven-year safety and efficacy with velaglucerase alfa for treatment-naïve adult patients with type 1 Gaucher disease
Ari Zimran, Nan Wang, Carol Ogg, et al.
Journal of Child Neurology
|
July 9, 2016
Metachromatic Leukodystrophy: An Assessment of Disease Burden
Florian S Eichler, Timothy M Cox, Eric Crombez, et al.
Blood Advances
|
April 8, 2025
Gene therapy for hemophilia B: results from the phase 1/2 101HEMB01/02 studies
Steven Pipe, Allen Poma, Anita Rajasekhar, et al.
Molecular Genetics and Metabolism
|
June 6, 2015
Long-term velaglucerase alfa treatment in children with Gaucher disease type 1 naïve to enzyme replacement therapy or previously treated with imiglucerase
Laurie Smith, William Rhead, Joel Charrow, et al.
American Journal of Hematology
|
March 18, 2015
Safety and efficacy results of switch from imiglucerase to velaglucerase alfa treatment in patients with type 1 Gaucher disease
Deborah Elstein, Atul Mehta, Derralynn A Hughes, et al.
American Journal of Hematology
|
February 7, 2013
Enzyme replacement therapy with velaglucerase alfa in Gaucher disease: Results from a randomized, double-blind, multinational, Phase 3 study
Derlis E Gonzalez, Hadhami Ben Turkia, Elena A Lukina, et al.
American Journal of Hematology
|
March 25, 2015
Velaglucerase alfa (VPRIV) enzyme replacement therapy in patients with Gaucher disease: Long-term data from phase III clinical trials
Derralynn A Hughes, Derlis E Gonzalez, Elena A Lukina, et al.
American Journal of Medical Genetics. Part A
|
October 22, 2008
Safety and efficacy of 22 weeks of treatment with sapropterin dihydrochloride in patients with phenylketonuria
Phillip Lee, Eileen P Treacy, Eric Crombez, et al.
American Journal of Hematology
|
February 13, 2013
Velaglucerase alfa enzyme replacement therapy compared with imiglucerase in patients with Gaucher disease
Hadhami Ben Turkia, Derlis E Gonzalez, Norman W Barton, et al.
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of 2
Search research articles
Search
Showing results (1-10 of 12) with videos related to
Sort By:
Page
of 2
The Journal of Pediatrics
|
July 20, 2005
Pitfalls in newborn screening
Eric Crombez, Richard Koch, Stephen Cederbaum
American Journal of Hematology
|
April 24, 2015
Seven-year safety and efficacy with velaglucerase alfa for treatment-naïve adult patients with type 1 Gaucher disease
Ari Zimran, Nan Wang, Carol Ogg, et al.
Journal of Child Neurology
|
July 9, 2016
Metachromatic Leukodystrophy: An Assessment of Disease Burden
Florian S Eichler, Timothy M Cox, Eric Crombez, et al.
Blood Advances
|
April 8, 2025
Gene therapy for hemophilia B: results from the phase 1/2 101HEMB01/02 studies
Steven Pipe, Allen Poma, Anita Rajasekhar, et al.
Molecular Genetics and Metabolism
|
June 6, 2015
Long-term velaglucerase alfa treatment in children with Gaucher disease type 1 naïve to enzyme replacement therapy or previously treated with imiglucerase
Laurie Smith, William Rhead, Joel Charrow, et al.
American Journal of Hematology
|
March 18, 2015
Safety and efficacy results of switch from imiglucerase to velaglucerase alfa treatment in patients with type 1 Gaucher disease
Deborah Elstein, Atul Mehta, Derralynn A Hughes, et al.
American Journal of Hematology
|
February 7, 2013
Enzyme replacement therapy with velaglucerase alfa in Gaucher disease: Results from a randomized, double-blind, multinational, Phase 3 study
Derlis E Gonzalez, Hadhami Ben Turkia, Elena A Lukina, et al.
American Journal of Hematology
|
March 25, 2015
Velaglucerase alfa (VPRIV) enzyme replacement therapy in patients with Gaucher disease: Long-term data from phase III clinical trials
Derralynn A Hughes, Derlis E Gonzalez, Elena A Lukina, et al.
American Journal of Medical Genetics. Part A
|
October 22, 2008
Safety and efficacy of 22 weeks of treatment with sapropterin dihydrochloride in patients with phenylketonuria
Phillip Lee, Eileen P Treacy, Eric Crombez, et al.
American Journal of Hematology
|
February 13, 2013
Velaglucerase alfa enzyme replacement therapy compared with imiglucerase in patients with Gaucher disease
Hadhami Ben Turkia, Derlis E Gonzalez, Norman W Barton, et al.
Page
of 2