Patients

Patient Resources

The following organizations provide educational resources and support research to help people living with rare, inherited diseases involving the liver and genetic cancers.

 

Rare Genetic Diseases of the Liver

“Global Genes is a leading rare disease advocacy organization dedicated to eliminating the challenges of rare diseases by spreading awareness, and providing education and resources to impact affected patients and their families.”

Dicerna is proud to be a member of the RARE Corporate Alliance™, which is “a collaborative group of rare disease stakeholders, including pharmaceutical companies, health-related organizations, industry and academia. The Alliance is committed to advancing efforts that benefit the rare disease community and its constituents.”

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“EURORDIS enables better lives and cures for people living with a rare disease. Its mission is to work across borders and diseases to improve the lives of people living with a rare disease.”

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“NORD provides advocacy, education and other services to improve the lives of all people affected by rare diseases.”

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“The Rare Disease United Foundation (RDUF) is the nation’s leading rare disease advocacy organization. RDUF works on the challenges facing rare disease patients both locally and nationally. In five years, RDUF has gone from the first state-based, non-disease specific non-profit to the voice of the 60 million people in the United States suffering from the physical, emotional and financial stress of living with a rare disease. RDUF programs target specific issues and approach them with the wisdom, knowledge and passion that comes with living rare.”

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Primary Hyperoxaluria

“The OHF is the only foundation in the world dedicated to improving the care and treatment and finding a cure for oxalosis, primary hyperoxaluria (including PH1) and related stone diseases.”

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“The PH Self-Help Association provides information about primary hyperoxaluria, patient testimonials, resources for patients and caregivers and updates on current research and clinical trials.”

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“AIRG-France was created in 1988 under the leadership of Professor Jean-Pierre Grünfeld and Dr. Ginette Albouze, some patients, their families and doctors anxious to establish networks to exchange the medical knowledge of genetic kidney diseases. The mission of this organization is to inform constituents about kidney genetic diseases and their consequences on the body and the life of patients as well as on organ donation and kidney transplantation.”

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“The Mayo Clinic Hyperoxaluria Center is a clinical care and research center staffed by physician scientists devoted to the study of primary hyperoxaluria. The goals of the center are to provide a resource for the diagnosis and care of patients with primary hyperoxaluria, to discover factors that predict or determine loss of kidney function in patients with primary hyperoxaluria, and to develop effective treatments.”

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“The Rare Kidney Stone Consortium facilitates cooperative exchange of information and resources among investigators, clinicians, and patients, and researchers in order to improve care and outcomes for patients with rare stone diseases.  The consortium promotes ready availability of diagnostic testing, pooling of clinical experiences, and availability of tissue banks in order to advance the science.”

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Additional Reading

 

Liver Disease

Rare Kidney Stone Consortium: Primary Hyperoxaluria

American Cancer Society: Risk Factors for Liver Disease

Review article: Viral hepatitis, non-alcoholic fatty liver disease and alcohol as risk factors for hepatocellular carcinoma (PDF)

This article reviews the viral characteristics associated with increased risk for development of hepatoceullular carcinoma (HCC) and the role of antiviral therapy in decreasing risk of HCC in those with viral hepatitis and cirrhosis. Association of alcoholic cirrhosis and non-alcoholic fatty liver disease with liver cancer will be reviewed as well as possible measures to decrease the risk of HCC in these highly prevalent populations.

This article provides an overview of Primary Hyperoxaluria type 1, including its epidemiology, different forms of the diseases and its genetic makeup. The article also presents information on the diagnosis, management and current treatment options for the condition, including transplantation and dialysis. Finally, the article provides preclinical data and possible therapeutic developments in the coming years as a result of research currently underway. Cochat, P, Rumsby, G. Primary hyperoxaluria. The New England Journal of Medicine 2013; 369(7): 649-658.