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A retrospective, multinational, multicenter study on the natural history of infantile-onset Pompe disease
The Journal of Pediatrics · 2006 · Vol. 148(5) · pp. 671–676.e2
Priya S. Kishnani✉(Duke Medical Center)Wuh‐Liang Hwu(Hôpital Debrousse)Hanna Mandel(Duke Medical Center)Marc Nicolino(Meyer Children's Hospital)Florence H. Yong(Meyer Children's Hospital)Deyanira Corzo(Meyer Children's Hospital)
Lysosomal Storage Disorders ResearchAcute Lymphoblastic Leukemia researchNeurogenetic and Muscular Disorders ResearchMedicinePediatricsHypotoniaRetrospective cohort studyFailure to thriveRespiratory distressProportional hazards modelNatural historyGlycogen storage disease type IISurvival analysis
MeSH terms
EuropeFemaleGlycogen Storage Disease Type IIHumansInfantInfant, NewbornIsraelMaleNorth AmericaRespiration, ArtificialRetrospective StudiesTaiwanCohort StudiesSurvival RateDisease Progression
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