A multicenter, open-label, phase III study of Abcertin in Gaucher disease

BACKGROUND: Gaucher disease (GD) is caused by a deficiency in the lysosomal enzyme glucocerebrosidase. Enzyme replacement therapy (ERT) is recommended for clinical improvement. METHODS: The efficacy and safety of a new imiglucerase, Abcertin, were assessed in 7 Egyptian patients with treatment-naïve...

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Udgivet i:Medicine (Baltimore)
Main Authors: Lee, Beom Hee, Abdalla, Ahmed Fathy, Choi, Jin-Ho, Beshlawy, Amal El, Kim, Gu-Hwan, Heo, Sun Hee, Megahed, Ahmed Megahed Hassan, Elsayed, Mona Abdel Latif, Barakat, Tarik El-Sayed Mohammad, Eid, Khaled Mohamed Abd El-Azim, El-Tagui, Mona Hassan, Mahmoud, Mona Mohamed Hamdy, Fateen, Ekram, Park, June-Young, Yoo, Han-Wook
Format: Artigo
Sprog:Inglês
Udgivet: Wolters Kluwer Health 2017
Fag:
5100
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5690733/
https://ncbi.nlm.nih.gov/pubmed/29137040
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1097/MD.0000000000008492
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