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Last Updated : 06/20/2022

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Found 2 publications

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
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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
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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