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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Detaylı Bibliyografya
Yayımlandı:Am J Hematol
Asıl Yazarlar: 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
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: Blackwell Publishing Ltd 2015
Konular:
Original Articles
Online Erişim: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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