NGF Community Blog

Everyone experiences inflammation, the body’s response to stress, illness, and other external inputs. People living with Gaucher disease may have a higher level of inflammatory response because their bodies are constantly working to manage the disease. The good news: You can help your body become more resilient. Dr. Robin Ely explains the role inflammation plays… Read More »

Stress always exists, but at times like now, amid the coronavirus pandemic, it’s a global phenomenon. As you watch events unfold in your communities and around the world, you aren’t alone if you feel lonely, anxious, and worried. In one way or another, everyone on the planet has been affected—directly or indirectly—by COVID-19, the disease… Read More »

Gaucher disease occurs when people have mutations, or changes, in a gene called GBA. The GBA gene acts like a switch that controls the way your body breaks down a specific lipid (fat), glucocerebroside. The “switch” is faulty in people with Gaucher disease. That prevents cells from making functional glucocerebrosidase (GCase), the enzyme that breaks… Read More »

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 »

Rare diseases can affect all aspects of a patient’s life, not just physical health. If you have Gaucher disease, it’s not just about finding a treatment that’s effective. Finding supportive resources, including peers who also live with the disease, can help you cope with your condition—and empower you to take charge of your health. Aviva… Read More »

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 »

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 »

Growth in Gaucher: Managing Bone Health Dr. Heather A. Lau, MD, MS, Assistant Professor and Director of the Lysosomal Storage Disorders Program at New York University School of Medicine, discusses managing bone health for those who have Gaucher disease with an emphasis on the impact early intervention and treatment, particularly when introduced before or during… Read More »

Illuminating Lyso-Gyl 1: Does the Key Biomarker of Gaucher Disease Hold the Key to Understanding? Dr. Pramod Mistry, MBBS, PhD, FRCP, Professor of Medicine, Pediatrics, and Cellular & Molecular Physiology, Department of Internal Medicine, Yale New Haven Hospital, discusses the importance of bio markers as they relate to patients with Gaucher disease. He illuminates the… Read More »

Medical research has always been important for furthering our understanding of disease. Through the completion of clinical studies, doctors and scientists identify new and effective treatment methods for some of the rarest diseases, including Gaucher disease. Gaucher Disease Clinical Trials The mainstay of Gaucher disease treatment—enzyme replacement therapy (ERT)—was developed thanks to clinical trials. More… Read More »