A tailor-made therapy, developed in a flash

(KATHERINE C. COHEN/BOSTON CHILDREN’S HOSPITAL)

Drug development is expensive — and it takes time. Which is what makes a recent case out of Boston Children's Hospital so extraordinary.

Faced with the case of a 6-year-old girl with Batten's disease, an ultra-rare neurodegenerative disorder, scientists and regulators made it from diagnosis to bespoke therapy in just over a year. Now that therapy, patterned on the same kind of treatment as Spinraza, which was recently approved to treat spinal muscular atrophy, may have helped to stop the disease in its tracks.

The case serves as a proof-of-concept in efforts to rapidly develop and deliver precision medicine — but it also may have involved a great deal of good fortune.

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