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Diseases

Genetic and Rare Diseases Information Center (GARD)

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Stargardt disease


Other Names for this Disease
  • Juvenile onset macular degeneration
  • Stargardt macular dystrophy
See Disclaimer regarding information on this site. Some links on this page may take you to organizations outside of the National Institutes of Health.


Your Question

Are there any dietary supplements or natural substances that may slow the progression of vision loss?

Our Answer

We have identified the following information that we hope you find helpful. If you still have questions, please contact us.

How might Stargardt disease be treated?

At present there is no cure for Stargardt disease, and there is very little that can be done to slow its progression. Wearing sunglasses to protect the eyes from UVa, UVb and bright light may be of some benefit. Animal studies have shown that taking excessive amounts of vitamin A and beta carotene could promote the additional accumulation of lipofuscin, as well as a toxic vitamin A derivative called A2E; it is typically recommended that these be avoided by individuals with Stargardt disease. There are possible treatments for Stargardt disease that are being tested, including a gene therapy treatment, which has been given orphan drug status by the European Medicines Agency (EMEA, similar to the FDA).[1] You can read more about this treatment by clicking here. There are also clinical trials involving embryonic stem cell treatments.[1]

The U.S. National Institutes of Health, through the National Library of Medicine, developed ClinicalTrials.gov to provide patients, family members, and members of the public with current information on clinical research studies. Currently, a number of clinical trials are identified as completed, active, or enrolling individuals with Stargardt disease and other types of macular degeneration. To find trials specific to Stargardt disease, click on the link above and use "Stargardt disease" as your search term, or click here. To find trials regarding all types of macular degeneration, use "macular degeneration" as your search term, or click here. After you click on a study, review its "eligibility" criteria to determine its appropriateness. Use the study’s contact information to learn more. Check this site often for regular updates.

Individuals interested in clinical trials can also contact the Patient Recruitment and Public Liaison (PRPL) Office at the National Institutes of Health (NIH). We recommend calling the toll-free number listed below to speak with a specialist, who can help you determine if you are eligible for any clinical trials. If you are located outside the United States, and would like to be contacted via telephone, you will need to provide your telephone number in full, including area code and international dialing prefix.

Patient Recruitment and Public Liaison Office
NIH Clinical Center
Bethesda, Maryland 20892-2655
Toll-free: 800-411-1222
Fax: 301-480-9793
Email: prpl@mail.cc.nih.gov
Web site:  http://clinicalcenter.nih.gov/

If you are interested in enrolling in a clinical trial, you can find helpful general information on clinical trials at the following ClinicalTrials.gov Web page.
http://clinicaltrials.gov/ct2/info/understand

Resources on many charitable or special-fare flights to research and treatment sites and low-cost hospitality accommodations for outpatients and family members, as well as ambulance services, are listed on the Web site of the Office of Rare Diseases Research (ORDR), part of the National Institutes of Health.
http://rarediseases.info.nih.gov/Resources.aspx?PageID=8
Last updated: 6/3/2011

References