WebThe National Gaucher Foundation (NGF) is an independent nonprofit dedicated to serving U.S. patients with Gaucher disease and their families. Through financial support, … WebApr 30, 2024 · People diagnosed with Gaucher disease typically require periodic tests to track its progression, including: Dual energy X-ray absorptiometry (DXA). This test uses …
What You Need to Know About Gaucher Disease Pfizer
WebApr 1, 2024 · Gaucher Disease (GD) is an inherited condition that can damage many different parts of the body. Damage occurs when a type of fat — glucocerebroside — … WebApr 1, 2024 · Gaucher Disease (GD) is an inherited condition that can damage many different parts of the body. Damage occurs when a type of fat — glucocerebroside — builds up in certain organs in the body. Normally, we have an enzyme—glucocerebrosidase — that eliminates this bad fat. People with GD do not make enough of this enzyme. chat box widget obs
National Gaucher Foundation
Gaucher (go-SHAY) disease is the result of a buildup of certain fatty substances in certain organs, particularly your spleen and liver. This causes these organs to enlarge and can affect their function. The fatty substances also can build up in bone tissue, weakening the bone and increasing the risk of fractures. If … See more There are different types of Gaucher disease, and signs and symptoms of disease vary widely, even within the same type. Type 1 is by far the most common. Siblings, even identical twins, with the disease can have … See more Gaucher disease can result in: 1. Delays in growth and puberty in children 2. Gynecological and obstetric problems 3. Parkinson's disease 4. Cancers such as myeloma, leukemia … See more Gaucher disease is passed along in an inheritance pattern called autosomal recessive. Both parents must be carriers of a Gaucher changed (mutated) gene for their child to inherit … See more People of Eastern and Central European Jewish (Ashkenazi) ancestry are at higher risk of developing the most common variety of Gaucher disease. See more WebMiglustat is used to treat Gaucher disease type 1 (a condition in which a certain fatty substance is not broken down normally in the body and instead builds up in some organs and causes liver, spleen, bone, and blood problems). Miglustat is in a class of medications called enzyme inhibitors. It works by preventing the body from producing the ... WebGaucher disease type 1 mostly affects the liver, spleen and bone marrow. The central nervous system is unaffected. It is the most common and least severe form of the disease. Signs of disease may occur any time between childhood and adulthood. Well-managed patients with Gaucher disease type 1 can expect to live a normal lifespan. chatbox widget obs