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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.
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] Among Ashkenazi Jews, the rate of carriers is considerably higher, at roughly one in 15. [40] Type II Gaucher's disease shows no particular preference for any ethnic group. [citation needed]
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.
Velaglucerase alfa, sold under the brand name Vpriv, is a medication used for the treatment of Gaucher disease Type 1. [1] It is a hydrolytic lysosomal glucocerebroside-specific enzyme, which is a recombinant form of glucocerebrosidase. It has an identical amino acid sequence to the naturally occurring enzyme. [2] It is manufactured by Shire plc.
Known also as Elelyso, taliglucerase won approval from the U.S. Food and Drug Administration in May 2012 as an orphan drug for the treatment of Type 1 Gaucher's disease. Protalix has licensed global development and commercialization rights for Elelyso to Pfizer, except for Brazil, where Protalix retains full rights.
It is a rare disease in dogs, with cats seven to ten times more likely to be infected. The disease in dogs can affect the lungs and skin, but more commonly the eye and central nervous system. [20] Ringworm is a fungal skin disease that in dogs is caused by Microsporum canis (70%), Microsporum gypseum (20%), and Trichophyton mentagrophytes (10% ...