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I have a brother who has been diagnosed with Best's Disease, and he has asked me (since he can no longer read a computer screen) to find out if there's an estimate in years for the cure. I wonder if you could please answer these questions: 

- Is there an estimate in years for a cure?

- Is there work being done in your country with stem cells, or inhibition of A2E-mediated toxicity (or other approaches)?

I would appreciate any information that you can give me - it would be great to give my brother some hope.  Thank you very much for taking the time to answer.  

Unfortunately, nobody in the whole world is able to answer your question. We don't know what will be the first way of treatment. It will probably be one of three :

  • Exchange of the defective gene by the wild- type gene.
  • Implantation of stem cells.
  • Medical treatment by metabolic medications.

 

In several countries in the world stem cells research is being conducted, including Israel.

I've been reading the information at your website. All 3 of my brothers have this disease.  Do you know if it comes down the maternal or paternal line?  So far, I haven't been diagnosed with it.  I'm due for my yearly exam.  Just thought you might find it interesting that 3 brothers in one family have the disease.

Beverly Ramsey

Best disease is almost always an autosomal dominant disease. This means that it can be transmitted from the father or the mother to their children. Statistically, half of the children will be affected. In a specific family, the number of affected descendants may be different. The fact that three boys are affected but no girl or girls is just a chance occurrence.

Regards,

Saul Merin, M.D.
Professor of Ophthalmology
Hadassah University Hospital

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