Early Treatment With Alglucosidase Alfa Prolongs Long-Term Survival of Infants With Pompe Disease

In a previous 52-wk trial, treatment with alglucosidase alfa markedly improved cardiomyopathy, ventilatory function, and overall survival among 18 children

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Bibliographische Detailangaben
Veröffentlicht in:	Pediatric research 2009-09, Vol.66 (3), p.329-335
Hauptverfasser:	Kishnani, Priya S, Corzo, Deya, Leslie, Nancy D, Gruskin, Daniel, van der Ploeg, Ans, Clancy, John P, Parini, Rosella, Morin, Gilles, Beck, Michael, Bauer, Mislen S, Jokic, Mikael, Tsai, Chen-En, Tsai, Brian W H, Morgan, Claire, O'Meara, Tara, Richards, Susan, Tsao, Elisa C, Mandel, Hanna
Format:	Artikel
Sprache:	eng
Schlagworte:	Biological and medical sciences Carbohydrates (enzymatic deficiencies). Glycogenosis clinical-investigation Errors of metabolism General aspects Medical sciences Medicine Medicine & Public Health Metabolic diseases Pediatric Surgery Pediatrics
Online-Zugang:	Volltext
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Beschreibung
Zusammenfassung:	In a previous 52-wk trial, treatment with alglucosidase alfa markedly improved cardiomyopathy, ventilatory function, and overall survival among 18 children
ISSN:	0031-3998 1530-0447
DOI:	10.1203/PDR.0b013e3181b24e94