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Consensus Conference: A reappraisal of Gaucher disease - diagnosis and disease management algorithms

Type 1 (non neuronopathic) Gaucher disease was the first lysosomal storage disorder for which an effective enzyme replacement therapy was developed and it has become a prototype for treatments for related orphan diseases. There are currently four treatment options available to patients with Gaucher...

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מידע ביבליוגרפי
Main Authors: Mistry, Pramod K., Cappellini, Maria Domenica, Lukina, Elena, Özsan, Hayri, Pascual, Sara Mach, Rosenbaum, Hanna, Solano, Maria Helena, Spigelman, Zachary, Villarrubia, Jesús, Watman, Nora Patricia, Massenkeil, Gero
פורמט: Artigo
שפה:Inglês
יצא לאור: 2011
נושאים:
גישה מקוונת:https://ncbi.nlm.nih.gov/pmc/articles/PMC3058841/
https://ncbi.nlm.nih.gov/pubmed/21080341
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/ajh.21888
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