Gaucher disease type 1: the first experience of enzyme replacement therapy in pediatric practice in Moldova - case report.
Rattachement africain : gb, bd, md. Niveau de preuve : code pays fourni par la source.
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) decreased by almost 60% in 6 months of ERT, reaching absolutely normal dimensions after 9 months. The ERT with Imiglucerase was tolerated very well by the patient, showing a clear improvement of clinical symptoms after 4-6 months of therapy, hematological picture and splenomegaly solving. Even if the little patient had to come every 2 weeks for infusion, her quality of life improved a lot, being a totally happy child, going to school and having friends. The ERT should be initiated immediately after diagnosis to prevent the multisystem complications.
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