[Webinar] Parenting Adolescents with Gaucher Disease
On October 8, the National Gaucher Foundation hosted a webinar featuring Laurie Bailey, Clinical Research Manager at Cincinnati Children’s Hospital. You can watch the recording below. Toolkit Presentation Slides Transitions Growing and Managing Your Own Healthcare: What Every Teen Living with an LSD Needs to Know to Manage Their Healthcare – Dawn Laney, MS American… Read More »
Meet Our Ambassadors
The National Gaucher Foundation just announced our first Ambassador cohort. Meet the elite group below. We asked…”What inspires you to live a better today?” Kelly Burns Rhode Island “Choosing what I love, and what inspires me to live a better today, is family. Family has been behind a lot of major changes recently in my… Read More »
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 Gaucher Specialist: A Critical Member of Your Care Team
A Gaucher specialist is an expert in diagnosing and treating Gaucher disease. Having this specialist on your care team is essential to receiving the best treatment possible. NGF can help you find a Gaucher specialist.
The Links Between Gaucher Disease and Parkinson’s Disease (Podcast Recap)
Research into the link between Gaucher disease and Parkinson’s disease may provide answers to treatment for both conditions. Gaucher Disease and Parkinson’s Diseases: An Unexpected Connection As Gaucher disease research progresses, scientists are discovering stronger links between the condition and other neurological illnesses, like Parkinson’s disease. But there’s still much to learn about how, and… Read More »
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.
It’s All in the Family: How We Can Use our Heritage to Find a Cure for Parkinson’s Disease
Individuals with Gaucher disease and those with Parkinson’s disease can share a common link: mutations in the GBA gene. Identifying and evaluating people who carry a GBA mutation, whether they have Gaucher disease or PD or are related to someone living with one of these diseases, is vital to helping scientists learn more and develop… Read More »
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 was so good-looking that her cuteness was almost a caricature. Her blonde hair fell straight onto her shoulders, with bangs that were cut to reveal her forehead, accentuating her big blue eyes. Her nose was small and turned up ever so slightly. She was petite,… Read More »
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