Tag: Gaucher disease

First approved by the FDA in 2010, the medication known as VPRIV was originally manufactured by Shire plc. This article details the complex manufacturing history of VPRIV, explaining how it transitioned from its original developer to being produced by Takeda Pharmaceuticals today.

Initially developed by Genzyme and later acquired by Sanofi, venglustat is an investigational oral medication functioning as a substrate reduction therapy. This small-molecule compound has been explored for treating several rare lysosomal storage disorders, including Fabry and Gaucher disease, by targeting the synthesis of harmful glycosphingolipids.

Approximately one in 40,000 to 60,000 live births are affected by Gaucher disease, a rare genetic disorder requiring specialized treatment. For many, this includes long-term enzyme replacement therapy with Cerezyme. Knowing **how to administer Cerezyme** properly is crucial for patient safety and treatment efficacy.

Approved by the FDA in 2010, Vpriv (velaglucerase alfa) has over a decade of real-world experience as a long-term enzyme replacement therapy (ERT). It is used to treat Type 1 Gaucher disease in adults and children ages 4 and older.

Affecting approximately 1 in 40,000 to 60,000 live births, Gaucher disease is a rare genetic disorder caused by a deficient enzyme called glucocerebrosidase. The **cerezyme treatment** is an enzyme replacement therapy designed to manage the symptoms of this serious condition.