Category: Gaucher Disease Treatment

Gaucher Disease and Myeloma: What Patients and Families Should Know

Two thirds of Gaucher patients have some evidence of bone disease that is directly related to the disease. Gaucher bone disease may lead to serious orthopedic complications but it is not a cancerous condition. Doctors know that a much smaller number of people with GD can develop multiple myeloma, a malignancy of bone marrow plasma… Read More »

What Is Our Impact? – NGF 2019 Impact Report

In our 2019 Impact Report, we take a look at some of our major achievements in heightening awareness of Gaucher Disease. Read about how NGF has connected with nearly 2,000 individuals who then received screening or testing for Gaucher with no out-of-pocket expenses; how millions of people are engaging with our content online; and how… Read More »

Developing Enzyme Replacement Therapy – An Insider’s Perspective

Enzyme replacement therapy (ERT) was first approved for use by the U.S. Food and Drug Administration in 1991. This revolutionary therapy was the first discovered to correct the underlying enzyme deficiency that causes the non-neuronopathic symptoms of Gaucher disease types 1 and 3. Today, thousands of people living with Gaucher disease receive ERT infusions about… Read More »

Gaucher: SRT, ERT & Drug Interactions | National Gaucher Foundation

Gaucher Disease: Enzyme Replacement Therapy, Substrate Reduction Therapy & Potential Drug Interaction Side Effects Substrate reduction therapy (SRT) first debuted as an alternative method of treating Gaucher disease in 2003. At that time, miglustat (Zavesca®) was the only known oral medication capable of managing Gaucher disease symptoms to prevent the condition from worsening. In 2014,… Read More »

The Gaucher Specialist: A Critical Member of Your Care Team

A Gaucher specialist is an expert in diagnosing and treating Gaucher disease. Having this specialist on your care team is essential to receiving the best treatment possible. NGF can help you find a Gaucher specialist.

NGF honors the life of biotech legend, Henri Termeer

National Gaucher Foundation is saddened by the sudden passing of Henri A. Termeer, the former chief executive officer (CEO) of Genzyme Corp. Described by a former colleague as “the dean of the biotech community,” Henri’s contributions to lysosomal storage diseases including Gaucher disease and other rare diseases are immeasurable. May his memory be for a blessing.

ERTs and SRTs: How They Work & What is New

For those whose lives are touched by Gaucher disease types 1 and 3, ERT (enzyme replacement therapy) and SRT (substrate reduction therapy) are often part of their regular routines. But in 1991, when the FDA approved the first form of ERT, this breakthrough felt miraculous, radically easing the disease’s signs and symptoms. This was followed… Read More »

Gaucher Disease Pain Management

For many Gaucher patients, dealing with pain is one of the most difficult parts of living with the disease. Effectively treating pain requires finding its source, which isn’t always straightforward. A variety of medications and therapies can relieve pain for Gaucher patients, although certain painkillers carry a high risk of addiction. A Gaucher specialist can… Read More »

The 25th Anniversary of FDA Approval of ERT

In 1991, the U.S. Food and Drug Administration (FDA) approved the first treatment ever for Gaucher disease (pronounced go-SHAY). This treatment, called enzyme replacement therapy (ERT), replaces the enzyme missing in patients with Gaucher disease with a modified version of the enzyme. ERT helps the body break down glucocerebroside, a fatty chemical that accumulates in… Read More »

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