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Tay-Sachs, Canavan, Sandhoff, GM1 and related diseases

Farber Disease Research

Farber Disease Research Overview

Farber is a very rare, progressive, and potentially life-threatening disease which happens when a fat (lipid) called ceramide builds up in the body. The buildup causes a wide range of severity and symptoms, making diagnosis difficult. A blood test can confirm the disease, and more treatment options are needed 1.

For more information, see All About Lysosomal Storage Disorders

For information on participating in a study, please visit Studies Recruiting Patients.

 

Where are we with Farber Research?

There are currently no treatment options for Farber disease. The only treatments available may temporarily address some of the symptoms, but don’t prevent the disease from getting worse 2.

Natural History Study

A natural history study is currently being conducted to better understand the presentation, likely course, and impact of Farber disease. All patients diagnosed with Farber disease are eligible, including both those who have and have not undergone hematopoietic stem cell transplant (HSCT). Additionally, if you have had a loved one pass away who was diagnosed with Farber disease, their records are also eligible for this study and could help lead to a deeper understanding of the condition.

For more information about the trial, please visit clinicaltrials.gov and Studies Recruiting Patients.

 

Footnote Reference: 

  1. “Farber's Disease.” Genetic and Rare Diseases Information Center, U.S. Department of Health and Human Services, www.rarediseases.info.nih.gov/diseases/6426/farbers-disease.
  2. “Farber's Disease Information Page.” National Institute of Neurological Disorders and Stroke, American Journal of Diseases of Children. www.ninds.nih.gov/Disorders/All-Disorders/Farbers-Disease-Information-Page.

 

 

 

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