New Model Can Advance Treatments, Cures For Rare Diseases (Forbes)

FORBES | If your child suffered from a rare and incurable disease, what would you do to find a cure?

My former colleague and good friend Dr. Chip Chambers faced just such a challenge, and took the bull by the horns. Last month, Dr. Chambers organized a unique medical conference that should serve as a model for advancing research and treatment for newly discovered rare diseases. On November 11, 136 physicians and scientists from 16 countries came together in Bethesda, Maryland for the Inaugural International Conference on Deficiency of ADA2. The medical experts were joined by nearly 70 patients and family members from six countries who are affected by DADA2 (Deficiency of Adenosine Deaminase 2), providing a unique opportunity for shared learnings and global connections.

As far as we know this is the shortest time between a disease being first described in the medical literature and the convening of an international meeting of this scale—less than three years. Nearly every physician and researcher who in some way has touched this disease was present.

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