News

The Complicated Relationship between Gaucher Disease and Parkinsonism: Insights from a Rare Disease

Medical Advisory Board member, Dr. Ellen Sidransky, recently published an article on the relationship between Gaucher disease and Parkinsonism. Read the full article below. Click here >>

Sanofi Initiates Phase 2 Clinical Trial to Evaluate Therapy for Genetic Form of Parkinson’s Disease

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Sanofi Genzyme, the specialty care global business unit of Sanofi, today announced the start of a Phase 2 trial of an investigational oral therapy for patients with Parkinson’s disease who carry a single copy of a gene mutation that is the most common genetic risk factor for the disease. The trial will assess the… Read More »

Children’s National Health System Announces the Creation of First-of-its-Kind Rare Disease Institute

The National Gaucher Foundation applauds Children’s National Health System for its creation of the Children’s National Rare Disease Institute (CNRDI). Developing standards of care for patients with rare diseases is essential. Coupling this focus along with knowledge gathering and rare disease research will enhance the care this population receives. The NGF is committed to assisting… Read More »

Shire VPRIV Gaucher study

Are you an adult 18 – 65 years of age who has been diagnosed with Gaucher disease and are not currently receiving treatment (within the past 12 months)? If so, there is a new research protocol enrolling participants that may be for you. The research focuses on the possible improvement in bone involvement associated with… Read More »

Sanofi Genzyme Announces Torch Awards

SANOFI GENZYME – We are proud to announce the first annual Sanofi Genzyme TORCH Awards! The TORCH Awards are your opportunity to recognize individuals of all ages who have made a significant contribution to the Lysosomal Storage Disorder (LSD) community. The nomination process is simple. To nominate, you will be asked to submit your answer… Read More »

New York Conference Raises Awareness for Gaucher Disease

As a culmination of Gaucher Awareness Month, on October 30, 2016 the National Gaucher Foundation hosted its fourth annual NY symposium to present the latest research and community innovations, and bring the Gaucher community together. Click here to read the article featured in Tablet on the event.

Gaucher Disease and Parkinson: An Opportunity to Participate in a Study

Researchers have linked Gaucher disease to a slightly increased risk of having Parkinson disease (PD) later in life. This is also true for carriers, who do not have the disease but can pass it to their children. In the general population, individuals have a 2 to 4 percent chance of developing Parkinson disease past age 60…. Read More »

Sanofi Genzyme announces “Seven Tests for Managing Gaucher”

It’s important to monitor Gaucher disease with regular assessments. For Gaucher Awareness Month, Sanofi Genzyme has developed a website to help you better understand the tests that your doctor may use to help better manage your disease. Called “Seven Tests for Managing Gaucher,” the site contains information on Gaucher tests and disease management goals using… Read More »

NGF Mourns the Loss of Dr. Roscoe Brady

Summary: The National Gaucher Foundation today mourns the passing of Dr. Roscoe Owen Brady, M.D., a pioneer in medicine and the father of enzyme therapy for lysosomal diseases, including Gaucher disease. Rockville, MD, June 15, 2016 — The National Gaucher Foundation today mourns the passing of Roscoe Owen Brady, M.D. Dr. Brady’s foundational studies paved… Read More »

New Gaucher research protocol enrolling participants

Are you an adult 18 – 65 years of age who has been diagnosed with Gaucher disease and are not currently receiving treatment (within the past 12 months)? If so, there is a new research protocol enrolling participants that may be for you. The research focuses on the possible improvement in bone involvement associated with… Read More »

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