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Agalsidase Beta (Injection)

Treats Fabry disease.

What works?

Learn more about the effects of these drugs. The most reliable research is summed up for you in our featured article.

Agalsidase beta is used to treat Fabry disease which is an inherited disease caused by the lack of an enzyme called alpha-galactosidase A, in the body. This enzyme is necessary for your body. Agalsidase beta is available only with your doctor's prescription… Read more
Brand names include
Fabrazyme
Drug classes About this
Endocrine-Metabolic Agent

What works? Research summarized

Evidence reviews

Enzyme replacement therapy for Anderson‐Fabry disease

Anderson‐Fabry disease is a rare X‐linked recessive (inherited) lysosomal storage disease. Lysosomal disorders are triggered when a particular enzyme exists in too small an amount or is missing altogether; thus substances that should be broken down by the enzyme and recycled build up in the cell. This can cause a wide range of symptoms which affect the entire body. The kidneys, heart and brain can be affected and this can result in major illness and reduced life expectancy among affected individuals. Symptomatic males and females with Anderson‐Fabry disease have been described within the literature, although the appearance of clinical signs may be delayed and more variable among females.

A systematic review of the clinical effectiveness and cost-effectiveness of enzyme replacement therapies for Fabry's disease and mucopolysaccharidosis type 1

The aim of this review was to determine the clinical effectiveness and cost-effectiveness of the administration of intravenous enzyme replacement therapy (ERT) to symptomatic patients for the prevention of long-term damage and symptoms in Fabry's disease and in mucopolysaccharidosis type 1 (MPS1).

A systematic review of the clinical effectiveness and cost-effectiveness of enzyme replacement therapies for Fabry's disease and mucopolysaccharidosis type 1

OBJECTIVES: To determine the clinical effectiveness and cost-effectiveness of the administration of intravenous enzyme replacement therapy (ERT) to symptomatic patients for the prevention of long-term damage and symptoms in Fabry's disease and in mucopolysaccharidosis type 1 (MPS1).

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Summaries for consumers

Enzyme replacement therapy for Anderson‐Fabry disease

Anderson‐Fabry disease is a rare X‐linked recessive (inherited) lysosomal storage disease. Lysosomal disorders are triggered when a particular enzyme exists in too small an amount or is missing altogether; thus substances that should be broken down by the enzyme and recycled build up in the cell. This can cause a wide range of symptoms which affect the entire body. The kidneys, heart and brain can be affected and this can result in major illness and reduced life expectancy among affected individuals. Symptomatic males and females with Anderson‐Fabry disease have been described within the literature, although the appearance of clinical signs may be delayed and more variable among females.

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