THE SCIENCE BEHIND BPAN

BPAN 2016 Research Meeting Summary

Some key takeaway points from the meeting were:

  • Allison presented on how our understanding of what symptoms individuals with BPAN have is changing. By analyzing the medical records all of you have so graciously sent, we are learning that the spectrum of symptoms is much broader than we originally thought. You can see some key highlights in Allison’s presentation

  • Dr. Hogarth has identified a compound that acts on the BPAN biological pathway. This drug is FDA-approved for another purpose so we are hopeful that if it shows promising results in the lab and on test animals, then we may be able to quickly move on to clinical trials in individuals with BPAN.

  • Our lab and Dr. Papandreou’s research group in the UK are also testing a number of other compounds that will hopefully lead to clinical trials in the future.

  • Finally, we learned that there were many advantages to holding a research meeting in Portland, including coordination and efficient processing of biological samples. Therefore, we will likely be replicating this type of meeting in the future for other NBIA disorders.