NGF Community Blog

Gene Therapy for Gaucher Disease: AAV, Lentivirus, & 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 »

Topics: About Gaucher Disease

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 »

Topics: About Gaucher Disease, Gaucher Disease Treatment, Living With Gaucher Disease

Gaucher Disease: How Peer Support and Empowerment Can Help

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 »

Topics: About Gaucher Disease, Living With Gaucher Disease

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 »

Topics: About Gaucher Disease, Gaucher Disease Research, Gaucher Disease Treatment

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 »

Topics: About Gaucher Disease, Gaucher Disease Research, Gaucher Disease Treatment

[MEDTalks Video] Managing Bone Health

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 »

Topics: About Gaucher Disease, Gaucher Disease Research, Living With Gaucher Disease

[MEDTalks Video] Does the Key Biomarker of Gaucher Disease Hold the Key to Understanding?

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 »

Topics: About Gaucher Disease, Gaucher Disease Research

Clinical Trials & Gaucher Disease: Phases, Types, & What You Should Know

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 »

Topics: About Gaucher Disease, Gaucher Disease Research

Repealing the Affordable Care Act & The Effects It Can Have

What is the Affordable Care Act The Affordable Care Act (ACA) represents one of the most substantial changes to our nation’s healthcare policy since the creation of Medicare and Medicaid. The legislation was first introduced and approved by a Democratic congressional majority. The Obama administration signed the ACA into law on March 23, 2010. When… Read More »

Topics: Insurance

What You Should Know About At-Home Genetic and Ancestry Testing Kits

Genetic testing, including ancestry testing, has increased in popularity in recent years thanks to direct-to-consumer genetic testing kits. In the past, consumers had to go through healthcare providers for any type of genetic testing. Now, anyone can order an inexpensive DNA test online from one of several genetic testing companies. In 2018 alone, over 12… Read More »

Topics: About Gaucher Disease

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