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Orphan diseases: Bringing academia, industry, and government into the game

To make headway in translating the growing body of genomic knowledge into new treatments for orphan or rare diseases, we have to bring everyone – academia, industry, government, patients, etc. – into the game. (Navin Rajagopalan/Fotopedia)

“If you build it, he will come,” the ghosts of baseball players past tell a farmer in Field of Dreams. But it’s not that easy. To put people in the seats you have to have all of the right pieces: the right team, including players and managers; the right park, one that works for both the team and the fans; and a passion for being the best at the game.

In the field of rare diseases, not only are institutions like Children’s Hospital Boston stepping up to the plate, but industry and government are joining the game, bringing expertise, guidance and infrastructure. Together, they’re starting to turn basic biomedical discoveries – many made possible only through dedicated patients and their families – into lifesaving treatments.

The shape of the game came into clear view last week at the 2nd Annual Rare Disease Symposium at Children’s. This daylong event, hosted by Children’s Technology Innovation & Development Office (TIDO) and the Manton Center for Orphan Disease Research, united scientists, clinicians, regulators, funders and industry. It showcased not only the depth of the team Children’s has fielded for orphan disease work, but also facilities and support available through industrial and governmental partners to conduct translational research on orphan diseases.

Without a doubt, the word of the day was genomes.

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