Outcome of Patients with Classical Infantile Pompe Disease Receiving Enzyme Replacement Therapy in Germany

Purpose: Enzyme replacement therapy (ERT) has been shown to improve outcome in classical infantile Pompe disease. The purpose of this study was to assess mortality, morbidity, and shortcomings of ERT in a larger cohort of patients treated outside clinical trials. To accomplish this, we retrospective...

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Pubblicato in:JIMD Rep
Autori principali: Hahn, Andreas, Praetorius, Susanne, Karabul, Nesrin, Dießel, Johanna, Schmidt, Dorle, Motz, Reinald, Haase, Claudia, Baethmann, Martina, Hennermann, Julia B., Smitka, Martin, Santer, René, Muschol, Nicole, Meyer, Ann, Marquardt, Thorsten, Huemer, Martina, Thiels, Charlotte, Rohrbach, Marianne, Seyfullah, Gökce, Mengel, Eugen
Natura: Artigo
Lingua:Inglês
Pubblicazione: Springer Berlin Heidelberg 2015
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Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC4375117/
https://ncbi.nlm.nih.gov/pubmed/25626711
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/8904_2014_392
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