Tag: Gaucher disease

Affecting millions worldwide, lysosomal storage disorders are rare genetic conditions caused by the accumulation of metabolic substrates within cells. The oral medication miglustat was developed to treat these conditions by tackling the root cause of the buildup, offering a versatile therapeutic approach.

Over 400 different genetic mutations have been identified that can lead to a deficiency of glucocerebrosidase, causing the lysosomal storage disorder known as Gaucher disease. This inherited condition prevents the body from properly breaking down a fatty substance called glucocerebroside, leading to its accumulation in cells and causing a wide range of health problems.

Inherited deficiency of the enzyme **Glucocerebrosidase** is the cause of Gaucher disease, a devastating lysosomal storage disorder that affects approximately 1 in 50,000 to 1 in 100,000 people worldwide. This crucial enzyme is known by several other names in the scientific and medical communities, reflecting its function and its genetic basis.

Affecting approximately 1 in 40,000 to 60,000 births, Gaucher disease is the most common lysosomal storage disorder, caused by a deficiency of the enzyme glucocerebrosidase (GCase). Understanding the specific therapeutic strategies for this genetic condition is essential for managing symptoms and improving quality of life, which is precisely how you treat glucocerebrosidase deficiency.

Without insurance, a single vial of Vpriv (velaglucerase alfa) has an approximate list price of nearly $1,500. As a treatment for the rare genetic disorder Gaucher disease, the annual cost can potentially exceed six figures, making the question of **how much does Vpriv cost?** a critical concern for patients and their families.

VPRIV (velaglucerase alfa) was approved by the U.S. Food and Drug Administration (FDA) in 2010 for long-term treatment of Type 1 Gaucher disease. For those asking what is the generic for VPRIV, it is important to understand that this medication, as a biologic, does not have a generic version in the traditional sense.

For patients with type 1 Gaucher disease, VPRIV is typically administered as a 60-minute intravenous infusion once every other week. However, the total time spent at an infusion center is often longer to account for patient assessment, medication preparation, and a crucial period of post-treatment monitoring by a healthcare professional.

Gaucher disease affects an estimated 1 in 50,000 to 100,000 people in the general population [1.10.4]. For those undergoing enzyme replacement therapy, a primary question is: **how long does a Cerezyme infusion take?** This intravenous treatment typically lasts between one to two hours [1.2.1, 1.2.2].

For the average patient, the annual list price of Cerezyme (imiglucerase) is approximately $200,000, though this figure can vary significantly based on dosage, disease severity, and individual factors. This high cost is typical for specialized orphan drugs designed to treat rare genetic disorders like Gaucher disease.

Over 6,000 patients worldwide are part of the International Collaborative Gaucher Group Gaucher Registry, which has provided long-term data supporting the use of Cerezyme (imiglucerase). This therapy is specifically approved for treating certain types of Gaucher disease and targets the debilitating symptoms associated with the condition. Understanding **what are the indications for Cerezyme** is crucial for patients and healthcare providers managing this rare genetic disorder.