What are Possible Future Developments?

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(From the Brochure)

Much remains to be learned about cystinosis. Investigators have recently isolated the gene causing cystinosis, and are now analyzing individual patients mutations. Other investigators are trying to determine the best therapies for each complication. Some questions still remain include:
  • Will children with cysteamine from infancy be spared all of the later complications of cystinosis?

  • Will they avoid kidney transplantation entirely?

  • Will cysteamine benefit patients who begin therapy after receiving a kidney tranplant?

  • Can cysteamine eye drops be approved by the Food and Drug Administration?

  • Will scientists discover the precise mechanism by which the cystinosis gene product rids lysosomes of cystine?

William A. Gahl, M.D., Ph.D.
National Institutes of Health
Bethesda, Maryland
1998 Revision

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