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Local boy with Duchenne muscular dystrophy begins promising gene-targeted therapy

Seven-year-old Wyatt Hubbard was a bit too focused on his right arm and its brief encounter with a needle to fully grasp the bigger picture. The needle was the first step to begin the infusion of a therapy just approved by the FDA for Duchenne muscular dystrophy (DMD), a rare and devastating disease that affects boys.

Wyatt became the first DMD patient in Ohio and undoubtedly one of the first in the country to receive Exondys 51 (eteplirsen) since it became available on the commercial market. After showing great promise in a clinical trial, it was granted accelerated approval by the FDA in September 2016.