Effect of Oral Eliglustat vs Placebo on Spleen Volume in Patients with Splenomegaly and Gaucher Disease Type 1: The ENGAGE Randomized Clinical Trial

IMPORTANCE: In Gaucher disease type 1, inherited deficiency of acid-β-glucosidase underlies accumulation of glucosylceramide in lysosomes of macrophages and resultant hepatosplenomegaly, anemia, thrombocytopenia, and skeletal disease. The standard of care is lifelong intravenous enzyme replacement t...

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Detalhes bibliográficos
Publicado no:JAMA
Main Authors: Mistry, Pramod K., Lukina, Elena, Turkia, Hadhami Ben, Amato, Dominick, Baris, Hagit, Dasouki, Majed, Ghosn, Marwan, Mehta, Atul, Packman, Seymour, Pastores, Gregory, Petakov, Milan, Assouline, Sarit, Balwani, Manisha, Danda, Sumita, Hadjiev, Evgueniy, Ortega, Andres, Shankar, Suma, Solano, Maria Helena, Ross, Leorah, Angell, Jennifer, Peterschmitt, M. Judith
Formato: Artigo
Idioma:Inglês
Publicado em: 2015
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4962880/
https://ncbi.nlm.nih.gov/pubmed/25688781
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1001/jama.2015.459
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