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The National Gaucher Foundation (United States) states the incidence of Gaucher's disease is about one in 20,000 live births. [39] Around one in 100 people in the general US population is a carrier for type I Gaucher's disease, giving a prevalence of one in 40,000. [ 40 ]
Eliglustat, sold under the brand name Cerdelga, is a medication used for the treatment of Gaucher's disease. It was discovered at the University of Michigan, developed by Genzyme Corp, and was approved by the FDA in August 2014. [7] Commonly used as the tartrate salt, the compound is believed to work by inhibition of glucosylceramide synthase.
Imiglucerase is a medication used in the treatment of Gaucher's disease. [2] [3]It is a recombinant DNA-produced analogue of the human enzyme β-glucocerebrosidase. Cerezyme is a freeze-dried medicine containing imiglucerase, manufactured by Genzyme Corporation.
The hematology specialist officially diagnosed me with Gaucher disease. The bone marrow biopsy confirmed my diagnosis of Gaucher disease—given that it’s genetic, it showed up in my DNA on the ...
ERT was not used in clinical practice until 1991, after the FDA gave orphan drug approval for the treatment of Gaucher disease with Alglucerase. [1] ERTs were initially manufactured by isolating the therapeutic enzyme from human placenta. [1]
Enzyme replacement therapy is available to treat mainly Fabry disease and Gaucher disease, and people with these types of sphingolipidoses may live well into adulthood. The other types are generally fatal by age 1 to 5 years for infantile forms, but progression may be mild for juvenile- or adult-onset forms.
Miglustat is indicated to treat adults with mild to moderate type I Gaucher disease for whom enzyme replacement therapy is unsuitable. [14]In the European Union, miglustat (Opfolda), in combination with cipaglucosidase alfa, is a long-term enzyme replacement therapy in adults with late-onset Pompe disease (acid α‑glucosidase [GAA] deficiency).
Taliglucerase alfa, sold under the brand name Elelyso among others, is a biopharmaceutical medication developed by Protalix and Pfizer. [3] [4] [full citation needed] The drug, a recombinant glucocerebrosidase used to treat Gaucher's disease, is the first plant-made pharmaceutical to win approval by the U.S. Food and Drug Administration (FDA).
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