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Last Updated : 06/20/2022
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Found 2 publications
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Efficacy, safety profile, and immunogenicity of alglucosidase alfa produced at the 4,000-liter scale in US children and adolescents with Pompe disease: ADVANCE, a phase IV, open-label, prospective study.
Condition:
Pompe disease
Journal:
Genetics in medicine : official journal of the American College of Medical Genetics
Treatment Used:
4000 liter scale alglucosidase
Number of Patients:
113
Published:
March 23, 2018
View publication
Summary:
Efficacy, safety profile, and immunogenicity of alglucosidase alfa produced at the 4,000-liter scale in US children and adolescents with Pompe disease: ADVANCE, a phase IV, open-label, prospective study.
High dose IVIG successfully reduces high rhGAA IgG antibody titers in a CRIM-negative infantile Pompe disease patient.
Condition:
CRIM-Negative Infantile Pompe Disease
Journal:
Molecular genetics and metabolism
Treatment Used:
High Dose Intravenous Immunoglobulin (IVIG)
Number of Patients:
1
Published:
June 27, 2017
View publication
Summary:
High dose IVIG successfully reduces high rhGAA IgG antibody titers in a CRIM-negative infantile Pompe disease patient.
Showing 1-2 of 2
Last Updated : 06/20/2022