Scientific Progress

    In his excellent paper to the Congress, Professor Gerry Chader, Chief Scientific Officer of the American Foundation Fighting Blindness, outlined the basic steps research needs to follow. These are:

    1. Accurate clinical evaluation of the patient (phentoyping).
      This is a necessary basis for any treatment or therapy. Get one and make sure you and your doctor are up to date on research progress. What you need to know - the exact type of retinal degeneration which you have, e.g. Macular Degeneration is not enough, is it Stargardt, Age-related Wet or Dry, etc. What was the age of onset and diagnosis? What are the clinical features?

    2. The exact gene mutation causing your RD (genotyping).
      Is your DNA banked at our gene-tracking laboratory in Cape Town?

    3. Animal Studies.
      The genetics and biological ramifications of the condition must be first studied in a suitable animal model and potential therapies developed (not done in South Africa).

    4. Proof of Principle.
      Such therapies must be demonstrated to be a potentially viable and basic safety standards proved.

    5. Government Approval for Limited Phase 1 Clinical Trials must be sought.

    6. Phase 1 - Human Clinical Trials.
      These trials are usually conducted on a small group of humans to check safety.

    7. Phase 2 - Human Clinical Trials.
      Small numbers of patients to check effectiveness of therapy.

    8. Phase 3 - Human Clinical Trials.
      Larger number of patients to confirm effectiveness.


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