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Did the Temporary Shortage in Supply of Imiglucerase Have Clinical Consequences? Retrospective Observational Study on 34 Italian Gaucher Type I Patients

Background. Enzyme Replacement Therapy (ERT) is the standard of care in Gaucher disease. The effects of withdrawal or reduced doses are debated, thus a retrospective cohort study was conducted to investigate clinical and laboratory differences in 34 Gaucher type 1 patients experiencing an ERT dosage...

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Main Authors: Deroma, Laura, Sechi, Annalisa, Dardis, Andrea, Macor, Daniela, Liva, Giulia, Ciana, Giovanni, Bembi, Bruno
Formato: Artigo
Idioma:Inglês
Publicado: Springer Berlin Heidelberg 2012
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Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC3575042/
https://ncbi.nlm.nih.gov/pubmed/23430505
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/8904_2012_158
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