Early access to medicines scheme (EAMS) scientific opinion: Raxone (idebenone) to treat the decline of respiratory function in patients with Duchenne Muscular Dystrophy (DMD)

This positive scientific opinion was renewed for Raxone to treat decline of respiratory function in patients with DMD from the age of 10 years who are currently not taking glucocorticoids; based on the very high level of unmet need in this patient population.

Medicines and Healthcare products Regulatory Agency