Can Gaucher disease be cured?

Zavesca is one of the best current treatment for Gaucher disease type 1, but there is no cure. Some people only experience mild symptoms, but others can experience severe pain, especially in the bones and abdomen. Other symptoms are severe bruising, fatigue and Symptoms can appear at any age.

At what age is Gaucher disease diagnosed?

The symptoms of Gaucher disease can manifest at any time. Some individuals may not receive a diagnosis until adulthood. Early onset osteoporosis could be an indicator yet many healthcare providers are unfamiliar with Gaucher disease, leading to a lack of testing for the condition.

How rare is Gaucher disease?

In the general population, Gaucher disease affects roughly 1 person in every 40,000. However, its frequency can be as high as 1 person in every 450 among Jews of Ashkenazi (Eastern European) descent. Although it is present at birth, symptoms can appear at any age.

What gender is most affected by Gaucher disease?

The Gaucher Registry tracks the incidence of the condition worldwide. 54% of registered patients are female, and 46% are male. The average age of diagnosis is 17, with nearly one haf being diagnosed before the age of 10.

Can people with Gaucher disease have children?

If one parent has Gaucher Disease and the other parent does not have the disease, and is not a carrier, all children will inherit the Gaucher gene from the one parent with Gaucher Disease, and will themselves become carriers. However, none of them will themselves have Gaucher Disease.

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Home / Online Pharmacy / Zavesca

US Name:

Zavesca
Alternative Names:

Zavesca
Active Ingredients:

Miglustat
Brand Manufacturer:

Actelion
Manufacturer Location:

Switzerland
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Zavesca ^*

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All third-party products, company names, and logos are trademarks™ or registered® trademarks are and remain the property of their respective holders. Their use does not imply any affiliation with or endorsement by them. All references on this site to third-party trademarks are intended to constitute nominative fair use under applicable trademark laws.

Zavesca is used to treat type 1 Gaucher disease for patients who do not respond well to enzyme replacement therapy. It is also used to treat adults and children with Niemann-Pick type-C disease,

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Zavesca Brand	100mg 84 Capsules	- Zavesca – Brand, Zavesca, 100mg, 84, Capsules, 77007, Actelion, 2012 + 0	Our price: $6,815.00

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Description
Side Effects
Indications
FAQ
Video

Zavesca (miglustat) is an oral medication recommended for patients with type 1 Gaucher disease. It is usually prescribed to adults who suffer from mild-to-moderate forms of this disease. Zavesca can relieve the symptoms of type 1 Gaucher disease by improving the functions of the liver and spleen and reducing the size of these organs. It may also help improve blood counts, including hemoglobin and platelet counts. Zavesca may be used only when enzyme replacement therapy fails to produce desired results or is considered unsuitable for the patient. Gaucher disease is a rare, inherited metabolic disorder in which there is an accumulation of harmful quantities of certain fats (lipids – specifically the glycolipid glucocerebroside), throughout the body, especially within the bone marrow, spleen, and liver. Zavesca is also effective in managing Niemann-Pick type-C disease in adults and children. It can improve the symptoms associated with eye movements, swallowing ability, and intellectual function. Zavesca can also slow down the progression of this disease and prevent the worsening of symptoms. Niemann Pick disease type-C (NPC) is a genetic condition that progresses slowly. It is known for causing issues in the body’s ability to move cholesterol and other fatty materials (lipids) within cells. This results in the buildup of these substances in different tissues, such as the brain tissue causing damage to those areas.

Dosage

The dosage and regimen will be set by the prescribing doctor. The usual starting dose of Zavesca for the treatment of type 1 Gaucher disease is one capsule three times each day. For Niemann-Pick type-C disease, the recommended dosage is two capsules three times daily for adults and children above 12. Normally, doctors calculate the dose for children younger than 12 years based on their age and weight. Zavesca capsules should be taken whole with a full glass of water. It can be taken with or without food.

Storage

Follow our tips to prevent medication mishaps. Store Zavesca below 30˚C in a safe location away from children. Do not use after the expiry date printed on the package. Do not throw any unused medicine out or flush it into a drain or toilet. Rather return it to a pharmacy for proper disposal.

This text is for informational purposes only. Please consult a doctor or pharmacist before using any medication.

Read the information leaflet that comes with the medication.

Zavesca may cause a few serious side effects, including:

Peripheral neuropathy resulting in pain, numbness, tingling, or burning in the hands or feet
Hand tremors or shaky movements

See a doctor immediately if these side effects are noticed.

Most people who use Zavesca do not experience any negative side effects. Doctors prescribe Zavesca because they assess that the benefits that such treatment yields outweigh any likely unwanted effects.

Some of the other side effects that have been reported include:

Diarrhea
Weight loss
Low platelet count
Dizziness
Vision problems
Dry mouth
Memory loss
Indigestion and bloating
Loss of appetite

Not all side effects are listed here. If these or other unlisted symptoms persist or worsen, consult a healthcare provider or pharmacist.

Zavesca is used to treat two inherited conditions: type 1 Gaucher disease and Niemann-Pick type-C disease. These conditions affect the mechanisms involved in the metabolism of fat, resulting in the buildup of fatty compounds known as glycosphingolipids in the body.

Symptoms commonly seen in Gaucher disease include an enlarged liver and/or spleen, low red blood cell levels, decreased platelet counts, and bone abnormalities. Platelets play a role in clotting, so individuals with low platelet levels may experience bleeding issues. Gaucher disease has three forms, categorized based on the presence or absence of neurological complications. Inheritance of all forms follows a recessive pattern.

Niemann-Pick disease type-C (NPC) affects both psychiatric functions, as well as various internal organs. Symptoms can manifest at different times and progress independently. Visceral symptoms are commonly observed in younger individual while neurological and psychiatric symptoms tend to develop gradually over time, becoming more noticeable in older age groups.

Not all affected individuals experience all the symptoms. Some children may face life-threatening complications early on while others might have a milder form of the disease that goes unnoticed until adulthood.

When NPC emerges during adolescence or adulthood, it may present with neurological features, as seen in childhood onset cases. However, the progression rate is typically slower in these cases. While specific manifestations can vary, they may include conditions like cerebellar ataxia, dysarthria, dysphagia, cognitive issues and movement disorders such as dystonia or tremors.

Can Gaucher disease be cured?

Zavesca is one of the best current treatment for Gaucher disease type 1, but there is no cure. Some people only experience mild symptoms, but others can experience severe pain, especially in the bones and abdomen. Other symptoms are severe bruising, fatigue and Symptoms can appear at any age.
At what age is Gaucher disease diagnosed?

The symptoms of Gaucher disease can manifest at any time. Some individuals may not receive a diagnosis until adulthood. Early onset osteoporosis could be an indicator yet many healthcare providers are unfamiliar with Gaucher disease, leading to a lack of testing for the condition.
How rare is Gaucher disease?

In the general population, Gaucher disease affects roughly 1 person in every 40,000. However, its frequency can be as high as 1 person in every 450 among Jews of Ashkenazi (Eastern European) descent. Although it is present at birth, symptoms can appear at any age.
What gender is most affected by Gaucher disease?

The Gaucher Registry tracks the incidence of the condition worldwide. 54% of registered patients are female, and 46% are male. The average age of diagnosis is 17, with nearly one haf being diagnosed before the age of 10.
Can people with Gaucher disease have children?

If one parent has Gaucher Disease and the other parent does not have the disease, and is not a carrier, all children will inherit the Gaucher gene from the one parent with Gaucher Disease, and will themselves become carriers. However, none of them will themselves have Gaucher Disease.

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All orders for prescription medication on this site require a valid prescription. The information found on this site is for informational purposes only. Please consult your doctor or pharmacist before purchasing any medication. Please review the instructions included with the medication before starting your treatment. You can contact us with any questions regarding usage, side effects, or drug interactions at any time.
All third-party products, company names, and logos are trademarks™ or registered® trademarks are and remain the property of their respective holders. Their use does not imply any affiliation with or endorsement by them. All references on this site to third-party trademarks are intended to constitute nominative fair use under applicable trademark laws.

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