Velaglucerase alfa (VPRIV) enzyme replacement therapy in patients with Gaucher disease: Long-term data from phase III clinical trials

Type 1 Gaucher disease is an inherited lysosomal enzyme deficiency with variable age of symptom onset. Common presenting signs include thrombocytopenia, anemia, hepatosplenomegaly, bone abnormalities, and, additionally in children, growth failure. Fifty-seven patients aged 3–62 years at the baseline...

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Detalhes bibliográficos
Publicado no:Am J Hematol
Main Authors: Hughes, Derralynn A, Gonzalez, Derlis E, Lukina, Elena A, Mehta, Atul, Kabra, Madhulika, Elstein, Deborah, Kisinovsky, Isaac, Giraldo, Pilar, Bavdekar, Ashish, Hangartner, Thomas N, Wang, Nan, Crombez, Eric, Zimran, Ari
Formato: Artigo
Idioma:Inglês
Publicado em: Blackwell Publishing Ltd 2015
Assuntos:
Original Articles
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4654249/
https://ncbi.nlm.nih.gov/pubmed/25801797
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/ajh.24012
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