Author: Adina Moses

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 »

[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 »

[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 »

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 »

Stay in Touch with NGF

Learn about the latest research, news and events.