NGF Community Blog

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

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

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 »

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

Ambassador Spotlight: Lauren Edwards

What were the events leading up to your diagnosis? What first led me to my diagnosis was getting a required blood test prior to starting Accutane, an oral medication for acne. When my results arrived, I discovered that my iron levels were extremely low and that I was anemic; I was then encouraged to see… Read More »

Topics: About Gaucher Disease, Ambassador Spotlight

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