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Gaucher Disease Treatment Market, by Disease Type, Therapy Type

06-22-2018 03:31 PM CET | Health & Medicine

Press release from: Pharma & Healthcare Industry

Gaucher Disease Treatment Market, by Disease Type, Therapy Type

Gaucher disease is an autosomal recessive inherited metabolism disorder where a type of fat (lipid) called glucocerebroside is unable to degrade. Body synthesis enzyme called glucocerebrosidase, which breakdowns and reprocesses glucocerebroside. Gaucher disease is caused by mutations of a single gene called GBA, which leads to very low levels of glucocerebrosidase enzyme leading to low degradation of glucocerebroside. There are three types of Gaucher disease namely: type 1, type 2, and type 3. Type 1 is the most common type of Gaucher disease while Type 2 and 3 are not as common as type 1.

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Approval of novel drugs is expected to boost growth of Gaucher disease treatment market

U.S. Food and Drug Administration (FDA) has approved multiple drugs in last few years for the treatment of disease. In 2010, FDA approved Vpriv (velaglucerase alfa), an enzyme replacement therapy (ERT), for type 1 Gaucher disease. In 2012, Elelyso (taliglucerase alfa) and in 2014, Cerdelga (eliglustat) was approved by FDA for type 1 Gaucher disease. These drugs offer variety of options based on their requirements, in turn is expected to boost the growth of the market.

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Gaucher disease is recessive gene dominance disorder with massive clinical heterogeneity. Symptoms of the disease include painless splenomegaly, anemia, or thrombocytopenia, chronic fatigue, hepatomegaly, bone pain, and pathologic fractures. The disease also contributes to easy incidence of bruising and bleeding through the nose. Manifested in three sub-types, the disease results from the deficiency of the enzyme glucocerebrosidase. It is the most common lysosomal storage disorder and has an incidence of