Clinical Trial Shows Efficacy of Rare Disease Treatment


The data published analyzes findings from key studies evaluating taliglucerase alfa in patients with Gaucher disease.

Data recently published in the Orphanet Journal of Rare Diseases showed that long-term improvements and stability can be achieved with taliglucerase alfa in patients with Gaucher disease.

Taliglucerase alfa, commercially known as Eleyso, is developed by Protalix and Pfizer. It was approved by the U.S. Food and Drug Administration (FDA) in May 2012 for use in adults with Gaucher disease, and a supplemental new drug application (sNDA) for pediatric use was granted approval in September of that same year.

Gaucher disease is a genetic lysosomal storage disorder in which a deficiency of the glucocerebrosidase enzyme leads to a buildup of the lipid glucerebrosidase in the lysosomes. The accumulation become encorged Gaucher cells and can cause multisystemic damage in organs and tissues, especially the spleen, liver, bone, platelets, and hemoglobin.

For more information, please visit

Related Videos
male pharmacist using digital tablet during inventory in pharmacy | Image Credit: sofiko14 -
Pharmacist holding medicine box in pharmacy drugstore. | Image Credit: I Viewfinder -
Pharmacy Drugstore Checkout Cashier Counter | Image Credit: Gorodenkoff -
Medicine tablets on counting tray with counting spatula at pharmacy | Image Credit: sutlafk -
Capsules medicine and white medicine bottles on table | Image Credit: Satawat -
Human cell or Embryonic stem cell microscope background | Image Credit: Anusorn -
Concept of health care, pharmaceutical business, drug prices, pharmacy, medicine and economics | Image Credit: Oleg -
Biosimilar pharmaceutical drug bottle on blue background. | Image Credit: Carl -
© 2024 MJH Life Sciences

All rights reserved.