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

  title={A multicenter, open-label, phase III study of Abcertin in Gaucher disease},
  author={B. Lee and A. Abdalla and J. Choi and A. Beshlawy and G. Kim and S. Heo and A. Megahed and M. A. Elsayed and Tarik Barakat and Khaled M. Eid and Mona Eltagui and M. M. Mahmoud and E. Fateen and June-Young Park and Han-Wook Yoo},
  • B. Lee, A. Abdalla, +12 authors Han-Wook Yoo
  • Published 2017
  • Medicine
  • Medicine
  • 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 type 1 GD. Each patient was administered a biweekly 60 U/kg dose of Abcertin for 6 months. The primary endpoint was the change in hemoglobin concentration. The secondary endpoints were changes from… CONTINUE READING
    3 Citations
    Current and Emerging Pharmacotherapy for Gaucher Disease
    • P. Giraldo
    • Medicine
    • Clinical Reviews in Bone and Mineral Metabolism
    • 2019
    The road to biosimilars in rare diseases ‐ ongoing lessons from Gaucher disease
    Lysosomes as a therapeutic target
    • 33
    • PDF


    Long-term clinical outcomes in type 1 Gaucher disease following 10 years of imiglucerase treatment
    • 23
    • PDF
    Replacement therapy with imiglucerase for type 1 Gaucher's disease
    • 101
    Individualization of long-term enzyme replacement therapy for Gaucher disease
    • 105
    • PDF
    Revised recommendations for the management of Gaucher disease in children
    • 65