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Andersen Disease
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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
The purpose of the study was to evaluate 4,000 L alglucosidase alfa in US infantile- or late-onset Pompe disease patients up to 1 year old.
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
This case report describes a a 7-year-old patient with cross-reactive immunologic material (CRIM)-negative infantile Pompe disease (IPD) treated with high dose and high frequency intravenous immunoglobulin (IVIG).
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