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Achievement of Therapeutic Goals with Low-Dose Imiglucerase in Gaucher Disease: A Single-Center Experience

Gaucher disease, a lysosomal storage disorder, is a multisystem disorder with variable and unpredictable onset and severity. Disease-specific enzyme replacement therapy (ERT) has been shown to reverse or ameliorate disease-specific hepatosplenomegaly and anemia and thrombocytopenia. ERT also impacts...

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Detalles Bibliográficos
Autores principales: Tukan, Irina, Hadas-Halpern, Irith, Altarescu, Gheona, Abrahamov, Ayala, Elstein, Deborah, Zimran, Ari
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Hindawi Publishing Corporation 2013
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3830843/
https://www.ncbi.nlm.nih.gov/pubmed/24285960
http://dx.doi.org/10.1155/2013/151506