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pubmed:abstractText |
Pompe disease causes progressive, debilitating, and often life-threatening musculoskeletal, respiratory, and cardiac symptoms. Favorable outcomes with early intravenous enzyme-replacement therapy and alglucosidase alfa have been reported, but early clinical diagnosis before the development of severe symptoms has rarely been possible in infants.
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pubmed:publicationType |
Journal Article,
Research Support, Non-U.S. Gov't,
Clinical Trial, Phase II,
Clinical Trial, Phase III
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