Drug Pricing, a Complex Issue Affecting the Rare Disease Community
Orphan drugs are expensive, and recent changes to the Orphan Drug Act may up the price even more. Find out how to take action and make your voice heard.
Precision Medicine and the Implications for Gaucher Disease (Podcast Recap)
Precision medicine could help enhance and direct treatment for Gaucher disease. But it might be challenging to obtain detailed genetic information from people living with Gaucher disease.
The Founder Effect’s Influence on Jewish Genetic Diseases
Among groups of people worldwide, genetic variation plays a key role in the prevalence of certain genetic diseases like Gaucher disease. Genetic diseases can affect anyone, but in some groups, certain diseases are more common compared to others. This can be attributed to genetic drift, or how frequently various genotypes (genetic makeups) appear in populations of individuals.
WORLDSymposium Highlights Key Gaucher Disease Research Insights
In February 2018, attendees from around the world met in San Diego for the 14th annual WorldSymposium, a research conference focused exclusively on the latest discoveries related to lysosomal diseases. Doctors and scientists met for three days to present and discuss cutting-edge research findings that may help change the diagnostic and treatment process for a variety of lysosomal diseases, including Gaucher disease.
Rare Disease Awareness
Written by Wayne Rosenfield, PhD She was a beautiful child. She... Read more
Resiliency and Gaucher Disease: Promoting Positive Outlooks and Outcomes
There is no standard definition for “resiliency” as it applies... Read more
[Podcast] Healthcare Legislation: Where We Are, And What’s Coming Next
You're probably confused by the recent healthcare industry turmoil. In our second podcast, we learn more about how bills are passed and what's going on now.
[Podcast] Gaucher Disease and U.S. Health Insurance
This month’s premier podcast is focused on the basics of... Read more
National Gaucher Foundation’s CARE Programs
It’s no secret that living with Gaucher disease can be... Read more
NGF Mourns Loss of Rosina Papantonio
The NGF is profoundly saddened by the recent passing of... Read more
