Velaglucerase alfa, sold under the brand name Vpriv and manufactured by Shire plc, is a hydrolytic lysosomal glucocerebroside-specific enzyme, which is a recombinant form of glucocerebrosidase indicated as a long-term enzyme replacement therapy for those with Gaucher disease Type 1. It has an identical amino acid sequence to the naturally occurring enzyme. It was approved for use by the U.S. Food and Drug Administration (FDA) on February 26, 2010.The most common side effects include abdominal (belly) pain, headache, dizziness, bone pain, arthralgia (joint pain), back pain, infusion-related reactions, asthenia (weakness) or fatigue (tiredness), and pyrexia (fever) or increased body temperature.Velaglucerase alfa was approved for medical use in the European Union in August 2010. Medical uses
Velaglucerase alfa is indicated for long-term enzyme-replacement therapy (ERT) in people with type-1 Gaucher disease. Competitive products
Imiglucerase is Genzymes version of recombinant glucocerebrosidase. It is marketed globally under the trade name of Cerezyme. In addition, Protalix and Pfizer have received approval for marketing taliglucerase alpha in Israel, Brazil, Canada and the U.S. The companies are expected also to file for marketing authorization in Europe and elsewhere around the world. References
External links
"Velaglucerase alfa". Drug Information Portal. U.S. National Library of Medicine.
Developmental coordination disorder and muscle weakness are usually treated with physiotherapy or occupational therapy. Dietary restriction of triglycerides has also been useful. Nutritionists often work with medical professionals to design appropriate dietary treatments for their patients. Prognosis
Prognosis can vary heavily based on the severity of the neurological dysfunction. If treatment is initiated early in disease the neurologic sequelae may be reversed and further deterioration can be prevented. Long-term outlook is reasonably good for most people when diagnosed and treated early. A case study presented a female patient diagnosed at the age of 11. Despite the relatively late diagnosis, the patient married and at the age of 34, gave birth to a full-term healthy infant. Her medication included vitamin K 10 mg twice a week, beta-carotene 40,000 IU daily, vitamin A 10,000 IU daily, vitamin E 400 IU daily, vitamins B6 and B12, calcium, magnesium and eye drops.Prolonged vitamin deficiencies can further compromise health. Specifically, a prolonged vitamin E deficiency can lead to the development of limiting ataxia and gait disturbances. Some individuals may develop retinal degeneration and blindness. If left untreated, the condition may lead to death. Current Research
The primary goal with Abetalipoproteinemia research is focused on supplying the fat-soluble vitamins the body lacks. Previous research considered the short term use of intravenous infusion of vitamins A and E. The goal was to determine whether these infusions would delay or counteract the symptoms in patients.