As I mentioned before, our Wolfram syndrome International Registry and Clinical Study has been selected to participate in the NIH Global Rare Disease Registry Program. Fortunately, I have been appointed to serve as a steering committee member for this important initiative. We would like to accomplish the following goals through this program.
For patients and their families:
Increase awareness of their specific rare disease and facilitating accelerated therapeutic development.
For physicians and nurses:
—Facilitate contact with others who are dealing with the same issues and compare notes.
—Provide access to outcomes data as a benchmark for their patients progress.
—Provide awareness of treatment regimens that are being attempted and seek better outcomes for their patients through novel approaches.
—Provide access to treatment information so that the health care providers can see how often a treatment is used for the condition of their patient.
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